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http://www.ncbi.nlm.nih.gov/pubmed/21685517
1. Georgian Med News. 2011 May;(194):24-8. The psychological aspects of burning mouth syndrome. Kenchadze R(1), Iverieli M, Okribelashvili N, Geladze N, Khachapuridze N. Author information: (1)Tbilisi State Medical University, Department of Therapeutic Stomatology, Georgia. It should be emphasized that at the present stage there is no consensus achieved regarding the etiopathogenesis of BMS. Almost all researchers point to lots of factors, simultaneously participating in genesis and development of BMS and at the same time most of them agreed on one - psychological factors play a crucial role in formation and maintenance of painful sensations. The aim of the study was the identification of psychological or psychiatric deviations (changes) among the patients with BMS to perform an adequate differentiated therapy. Clinico-psychological examination (dentist, neurologist, psychiatrist) was carried out in 39 patients from 46 to 70 years of age. Among them women - 36 and men - 3. To identify clinical types of BMS a classification of P.J. Lamey (1996) was used and as a result, depression, insomnia, cancerophobia, severe neurologic disorders, phobic syndrome were revealed. Three main categories - a chronic somatoform dysfunction (23 cases), chronic vegetative disorders (8), and chronic pain phenomenon (12) were identified. Only in one case was revealed a paranoid syndrome. Alongside with the well-known scheme of treatment (antidepressants, anticonvulsants, or neuroleptics) Psychotherapy was conducted, while EEG-feed back (Biofeed back, Neurofeed back) method was used for the first time. A number of important decisions were made the most important of which are the following: BMS - must be regarded as a psychosomatic problem rather than a psychiatric disorder. In addition to psychotherapy, using of EEG - feedback method greatly improved patients' condition and in 4 cases BMS clinical manifestations were evened-out completely. PMID: 21685517 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/11441716
1. Ned Tijdschr Tandheelkd. 2001 Jun;108(6):237-41. [Burning mouth syndrome]. [Article in Dutch] van der Waal I(1). Author information: (1)Uit de afdeling Mondziekten en kaakchirurgie/Orale pathologie/Algemene ziekteleer van het Vrije Universiteit Medisch Centrum, Amsterdam/Academisch Centrum Tandheelkunde Amsterdam (ACTA). Symptoms of a burning sensation of the oral mucosa mainly occur in the elderly, more often in women than in men. Often accompanying symptoms are complaints of a dry mouth and taste disturbances, all together referred to as the burning mouth syndrome. In the majority of cases there is no detectable cause. Although a psychogenic aetiology has often been put forward, no scientific evidence has ever been provided on this matter. In the majority of patients the burning mouth syndrome will disappear spontaneously, although this may take many years. PMID: 11441716 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/17849966
1. J Calif Dent Assoc. 2007 Jun;35(6):397-404. The burning mouth. McDonald JS(1). Author information: (1)University of Oral Pathology Service, Cincinnati, Ohio 45219, USA. Burning in the mouth in and of itself is not all that uncommon. It may result from a variety of local or generalized oral mucosal disorders, or may be secondary to referred phenomena from other locations. Primary burning mouth syndrome, on the other hand, is relatively uncommon. Burning mouth syndrome is an idiopathic pain disorder, which appears to be neuropathic in origin. Thoughts on management of secondary and particularly primary burning mouth syndrome are discussed. PMID: 17849966 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/7838464
1. Oral Surg Oral Med Oral Pathol. 1994 Nov;78(5):590-3. doi: 10.1016/0030-4220(94)90169-4. Lip component of burning mouth syndrome. Lamey PJ(1), Lamb AB. Author information: (1)Pain Relief Foundation, School of Clinical Dentistry, Queen's University of Belfast, Royal Victoria Hospital, U.K. To our knowledge there has been no previous study of factors specifically involved in the pathogenesis of patients who complain of burning sensation of the lips when the lips appear clinically normal. The complaint is akin to patients who complain of a burning sensation of the mouth when it appears clinically normal, a condition known as burning mouth syndrome. This study therefore studied precipitating factors in patients with burning mouth syndrome who reported lip involvement. Previous studies have shown that the lips are the third most common site reported as involved in patients who have burning mouth syndrome. Indeed patients with burning mouth syndrome often report multiple oral site involvement. To investigate the precipitating factors involved in the lip component of burning mouth syndrome, we studied 104 patients who reported the lips as a site affected by the condition from a total population of 312 patients with burning mouth syndrome. Hematologic, biochemical, and microbiologic parameters were studied in these patients. Sialometry, patch testing, psychological testing, and examination of denture status as well as questioning of parafunctional habits were also undertaken. No clear differences were noted in relationship to the frequency of abnormalities in burning mouth syndrome alone or burning mouth syndrome with lip involvement suggesting that similar precipitating factors apply. The precipitating factors in patients with lip involvement were found to be the same as burning mouth syndrome in general. Treatment of patients with lip involvement alone in burning mouth syndrome or lip involvement in burning mouth syndrome in conjunction with other intraoral sites gave an equally good response.(ABSTRACT TRUNCATED AT 250 WORDS) DOI: 10.1016/0030-4220(94)90169-4 PMID: 7838464 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/15024358
1. Oral Surg Oral Med Oral Pathol Oral Radiol Endod. 2004 Mar;97(3):339-44. doi: 10.1016/j.tripleo.2003.09.017. Psychological profile in burning mouth syndrome. Al Quran FA(1). Author information: (1)Department of Restorative Dentistry, Faculty of Dentistry, Jordan University of Science and Technology, Irbid, Jordan. firasquran@hotmail.com Thirty-two patients with burning mouth syndrome and 32 matched control subjects were evaluated for their personality profile using a comprehensive, reliable, and validated inventory. All subjects were requested to complete the Neo PI-R questionnaire that measures the 5 dimensions of personality and their facets. A t-test and univariate correlations (Pearson's correlation coefficient) were used to compare the 2 groups. Results show high significant differences in some personality factors. Neuroticism and all its facets, which include anxiety, angry hostility, depression, self-consciousness, impulsiveness and vulnerability, were significant at P<.001. Other domains like extraversion, openness, and conscientiousness showed significant differences also (P<.05). Many personality characteristics differentiate burning mouth syndrome patients from controllers according to the Neo PI-R and this should affect the treatment plan according to the identified characteristics. DOI: 10.1016/j.tripleo.2003.09.017 PMID: 15024358 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/21903545
1. Gen Dent. 2011 May-Jun;59(3):210-20; quiz 221-2. Burning mouth syndrome: a challenge for dental practitioners and patients. Klasser GD(1), Epstein JB, Villines D, Utsman R. Author information: (1)Department of Oral Medicine and Diagnostic Sciences, Universitry of Illinois, Chicago College of Dentistry, USA. A retrospective study was conducted on patients with burning mouth syndrome (BMS) to assess demographics, onset characteristics, temporal behavior (frequency), duration, and progression of oral burning symptoms. Additionally, treatments provided by health practitioners prior to a definitive diagnosis of BMS were analyzed with an overview of current management strategies. The records of 49 adult patients diagnosed with BMS were reviewed. Descriptive statistics and a Pearson correlation with a statistical significance at p < 0.05 were utilized to analyze the data. The majority of patients were mid-life white women who reported a sudden onset of constant oral burning symptoms that increased in intensity. On average, patients reported oral burning symptoms for 41 months (standard deviation = 73.5, range = 2-360 months, median = 20 months), and 38 of the patients received/trialed 71 various interventions (mean = 1.9) prior to receiving a definitive diagnosis for their oral burning symptoms. This study sample shared many characteristics with those reported previously in the literature. The authors found that patients frequently reported delays in receiving a definitive diagnosis with an array of various trialed interventions. For this reason, the authors provide this overview of current management strategies in order to assist dental practitioners in providing appropriate interventions for patients with BMS. PMID: 21903545 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/19658340
1. Lijec Vjesn. 2002 Jun-Jul;124(6-7):220-4. [Burning mouth syndrome--etiologic, diagnostic and therapeutic considerations]. [Article in Croatian] Alajbeg I(1), Vucićević-Boras V. Author information: (1)Zavod za oralnu medicinu Stomatoloskog fakulteta u Zagrebu. Burning mouth syndrome represents a chronic orofacial pain disorder without an apparent lesion of oral mucosa. It affects 0.71-3.4% of general population. Although a lot of scientific and clinical effort has been undertaken, its genesis still remains an enigma. Potential etiologic roles of various oral and systemic factors have been implicated, such as oral candidiasis, hormonal, neurological, nutritive and psychiatric disorders. In spite of different treatment approaches and medications, therapeutic success is limited. Although in our country most of the patients are treated by dentists specialized in oral medicine, medical doctors should be more involved in diagnosis and treatment of the disease. Article describes etiologic factors, diagnostic and therapeutic means in the burning mouth syndrome. PMID: 19658340 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23713105
1. Pediatrics. 2013 Jun;131(6):e1996-2001. doi: 10.1542/peds.2012-0749. Epub 2013 May 27. Complex chromosome rearrangement of 6p25.3->p23 and 12q24.32->qter in a child with moyamoya. Rosenberg RE(1), Egan M, Rodgers S, Harter D, Burnside RD, Milla S, Pappas J. Author information: (1)Department of Pediatrics, New York University School of Medicine, New York, NY, USA. rebecca.rosenberg@nyumc.org A 7-year-old white girl presented with left hemiparesis and ischemic stroke secondary to moyamoya syndrome, a progressive cerebrovascular occlusive disorder of uncertain but likely multifactorial etiology. Past medical history revealed hearing loss and developmental delay/intellectual disability. Routine karyotype demonstrated extra chromosomal material on 6p. Single nucleotide polymorphism microarray revealed a previously unreported complex de novo genetic rearrangement involving subtelomeric segments on chromosomes 6p and 12q. The duplicated/deleted regions included several known OMIM-annotated genes. This novel phenotype and genotype provides information about a possible association of genomic copy number variation and moyamoya syndrome. Dosage-sensitive genes in the deleted and duplicated segments may be involved in aberrant vascular proliferation. Our case also emphasizes the importance of comprehensive evaluation of both developmental delay and congenital anomalies such as moyamoya. DOI: 10.1542/peds.2012-0749 PMID: 23713105 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/20068102
1. Clin Cancer Res. 2010 Jan 15;16(2):681-90. doi: 10.1158/1078-0432.CCR-09-1091. Epub 2010 Jan 12. Identification of markers of taxane sensitivity using proteomic and genomic analyses of breast tumors from patients receiving neoadjuvant paclitaxel and radiation. Bauer JA(1), Chakravarthy AB, Rosenbluth JM, Mi D, Seeley EH, De Matos Granja-Ingram N, Olivares MG, Kelley MC, Mayer IA, Meszoely IM, Means-Powell JA, Johnson KN, Tsai CJ, Ayers GD, Sanders ME, Schneider RJ, Formenti SC, Caprioli RM, Pietenpol JA. Author information: (1)Departments of Biochemistry, Vanderbilt-Ingram Cancer Center, Vanderbilt University Medical Center, Nashville, Tennessee 37232, USA. PURPOSE: To identify molecular markers of pathologic response to neoadjuvant paclitaxel/radiation treatment, protein and gene expression profiling were done on pretreatment biopsies. EXPERIMENTAL DESIGN: Patients with high-risk, operable breast cancer were treated with three cycles of paclitaxel followed by concurrent paclitaxel/radiation. Tumor tissue from pretreatment biopsies was obtained from 19 of the 38 patients enrolled in the study. Protein and gene expression profiling were done on serial sections of the biopsies from patients that achieved a pathologic complete response (pCR) and compared to those with residual disease, non-pCR (NR). RESULTS: Proteomic and validation immunohistochemical analyses revealed that alpha-defensins (DEFA) were overexpressed in tumors from patients with a pCR. Gene expression analysis revealed that MAP2, a microtubule-associated protein, had significantly higher levels of expression in patients achieving a pCR. Elevation of MAP2 in breast cancer cell lines led to increased paclitaxel sensitivity. Furthermore, expression of genes that are associated with the basal-like, triple-negative phenotype were enriched in tumors from patients with a pCR. Analysis of a larger panel of tumors from patients receiving presurgical taxane-based treatment showed that DEFA and MAP2 expression as well as histologic features of inflammation were all statistically associated with response to therapy at the time of surgery. CONCLUSION: We show the utility of molecular profiling of pretreatment biopsies to discover markers of response. Our results suggest the potential use of immune signaling molecules such as DEFA as well as MAP2, a microtubule-associated protein, as tumor markers that associate with response to neoadjuvant taxane-based therapy. DOI: 10.1158/1078-0432.CCR-09-1091 PMCID: PMC2892225 PMID: 20068102 [Indexed for MEDLINE] Conflict of interest statement: Disclosure of Potential Conflicts of Interest A.B. Chakravarthy: commercial research grants for the support of the clinical trials from Aventis and BMS. The other authors disclosed no potential conflicts of interest.
http://www.ncbi.nlm.nih.gov/pubmed/19485423
1. J Proteome Res. 2009 Jul;8(7):3430-8. doi: 10.1021/pr900071h. Identification of differentially expressed proteins in triple-negative breast carcinomas using DIGE and mass spectrometry. Schulz DM(1), Böllner C, Thomas G, Atkinson M, Esposito I, Höfler H, Aubele M. Author information: (1)Institute of Pathology, Helmholtz Zentrum München, German Research Center for Environmental Health, Ingolstadter Landstrasse 1, 85764 Neuherberg, Germany. We compared the protein expression pattern of triple-negative breast carcinomas (HER2-, ER-, PR-) versus those being positive for HER2 and negative for the hormone receptors (HER2+, ER-, PR-) by 2-D DIGE and mass spectrometry. We obtained differential expression patterns for several glycolytic enzymes (as for example MDH2, PGK1, TKT, Aldolase1), cytokeratins (CK7, 8, 9, 14, 17, 19), further structure proteins (vimentin, fibronectin, L-plastin), for NME1-NME2, lactoferrin, and members of the Annexin family. Western blot analysis and immunohistochemistry were conducted to verify the results. The identified marker proteins may advance a more detailed characterization of triple-negative breast cancers and may contribute to the development of better treatment strategies. DOI: 10.1021/pr900071h PMID: 19485423 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/21418666
1. BMJ Clin Evid. 2010 Jul 19;2010:1301. Burning mouth syndrome. Buchanan JA(1), Zakrzewska JM. Author information: (1)Barts and The London School of Medicine and Dentistry, Dental Institute, Royal London Hospital, London, UK. INTRODUCTION: Burning mouth syndrome mainly affects women, particularly after the menopause, when its prevalence may be 18% to 33%. METHODS AND OUTCOMES: We conducted a systematic review and aimed to answer the following clinical question: What are the effects of treatments for burning mouth syndrome? We searched: Medline, Embase, The Cochrane Library, and other important databases up to November 2009 (Clinical Evidence reviews are updated periodically, please check our website for the most up-to-date version of this review). We included harms alerts from relevant organisations such as the US Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). RESULTS: We found 15 systematic reviews, RCTs, or observational studies that met our inclusion criteria. We performed a GRADE evaluation of the quality of evidence for interventions. CONCLUSIONS: In this systematic review we present information relating to the effectiveness and safety of the following interventions: anaesthetics (local), antidepressants, benzodiazepines (topical clonazepam), benzydamine hydrochloride, cognitive behavioural therapy (CBT), dietary supplements, and hormone replacement therapy (HRT) in postmenopausal women. PMCID: PMC3217740 PMID: 21418666 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/10478959
1. J Oral Pathol Med. 1999 Sep;28(8):350-4. doi: 10.1111/j.1600-0714.1999.tb02052.x. Burning mouth syndrome: prevalence and associated factors. Bergdahl M(1), Bergdahl J. Author information: (1)Department of Odontology, Umeå University, Sweden. Burning mouth syndrome (BMS) is characterized by a burning sensation in the oral cavity although the oral mucosa is clinically normal. The syndrome mostly affects middle-aged women. Various local, systemic and psychological factors have been found to be associated with BMS, but its etiology is not fully understood. Oral complaints and salivary flow were surveyed in 669 men and 758 women randomly selected from 48,500 individuals between the ages 20 and 69 years. Fifty-three individuals (3.7%), 11 men (1.6%) and 42 women (5.5%), were classified as having BMS. In men, no BMS was found before the age group 40 to 49 years where the prevalence was 0.7%, which increased to 3.6% in the oldest age group. In women, no BMS was found in the youngest age group, but in the age group 30 to 39 years the prevalence was 0.6% and increased to 12.2% in the oldest age group. Subjective oral dryness, age, medication, taste disturbances, intake of L-thyroxines, illness, stimulated salivary flow rate, depression and anxiety were factors associated with BMS. In individuals with BMS, the most prevalent site with burning sensations was the tongue (67.9%). The intensity of the burning sensation was estimated to be 4.6 on a visual analogue scale. There were no increased levels of depression, anxiety or stress among individuals with more pain compared to those with less pain. It was concluded that BMS should be seen as a marker of illness and/or distress, and the complex etiology of BMS demands specialist treatment. DOI: 10.1111/j.1600-0714.1999.tb02052.x PMID: 10478959 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22934887
1. J Proteome Res. 2012 Oct 5;11(10):5034-45. doi: 10.1021/pr300606e. Epub 2012 Sep 20. Mass spectrometry (LC-MS/MS) identified proteomic biosignatures of breast cancer in proximal fluid. Whelan SA(1), He J, Lu M, Souda P, Saxton RE, Faull KF, Whitelegge JP, Chang HR. Author information: (1)Department of Biochemistry, School of Medicine, Boston University, Boston, Massachusetts, USA. We have begun an early phase of biomarker discovery in three clinically important types of breast cancer using a panel of human cell lines: HER2 positive, hormone receptor positive and HER2 negative, and triple negative (HER2-, ER-, PR-). We identified and characterized the most abundant secreted, sloughed, or leaked proteins released into serum free media from these breast cancer cell lines using a combination of protein fractionation methods before LC-MS/MS mass spectrometry analysis. A total of 249 proteins were detected in the proximal fluid of 7 breast cancer cell lines. The expression of a selected group of high abundance and/or breast cancer-specific potential biomarkers including thromobospondin 1, galectin-3 binding protein, cathepsin D, vimentin, zinc-α2-glycoprotein, CD44, and EGFR from the breast cancer cell lines and in their culture media were further validated by Western blot analysis. Interestingly, mass spectrometry identified a cathepsin D protein single-nucleotide polymorphism (SNP) by alanine to valine replacement from the MCF-7 breast cancer cell line. Comparison of each cell line media proteome displayed unique and consistent biosignatures regardless of the individual group classifications, demonstrating the potential for stratification of breast cancer. On the basis of the cell line media proteome, predictive Tree software was able to categorize each cell line as HER2 positive, HER2 negative, and hormone receptor positive and triple negative based on only two proteins, muscle fructose 1,6-bisphosphate aldolase and keratin 19. In addition, the predictive Tree software clearly identified MCF-7 cell line overexpresing the HER2 receptor with the SNP cathepsin D biomarker. DOI: 10.1021/pr300606e PMCID: PMC3521600 PMID: 22934887 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/12766776
1. Nat Cell Biol. 2003 Jun;5(6):566-71. doi: 10.1038/ncb996. Rheb promotes cell growth as a component of the insulin/TOR signalling network. Saucedo LJ(1), Gao X, Chiarelli DA, Li L, Pan D, Edgar BA. Author information: (1)Division of Basic Sciences, Fred Hutchinson Cancer Research Center, 1100 Fairview Avenue North, Seattle, WA 98109, USA. Erratum in Nat Cell Biol. 2003 Jul;5(7):680. Insulin signalling is a potent stimulator of cell growth and has been proposed to function, at least in part, through the conserved protein kinase TOR (target of rapamycin) [corrected]. Recent studies suggest that the tuberous sclerosis complex Tsc1-Tsc2 may couple insulin signalling to Tor activity [corrected]. However, the regulatory mechanism involved remains unclear, and additional components are most probably involved. In a screen for novel regulators of growth, we identified Rheb (Ras homologue enriched in brain), a member of the Ras superfamily of GTP-binding proteins. Increased levels of Rheb in Drosophila melanogaster promote cell growth and alter cell cycle kinetics in multiple tissues. In mitotic tissues, overexpression of Rheb accelerates passage through G1-S phase without affecting rates of cell division, whereas in endoreplicating tissues, Rheb increases DNA ploidy. Mutation of Rheb suspends larval growth and prevents progression from first to second instar. Genetic and biochemical tests indicate that Rheb functions in the insulin signalling pathway downstream of Tsc1-Tsc2 and upstream of TOR. Levels of rheb mRNA are rapidly induced in response to protein starvation, and overexpressed Rheb can drive cell growth in starved animals, suggesting a role for Rheb in the nutritional control of cell growth. DOI: 10.1038/ncb996 PMID: 12766776 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22178447
1. J Proteomics. 2012 Jun 6;75(10):3031-40. doi: 10.1016/j.jprot.2011.11.033. Epub 2011 Dec 8. Stat1 and CD74 overexpression is co-dependent and linked to increased invasion and lymph node metastasis in triple-negative breast cancer. Greenwood C(1), Metodieva G, Al-Janabi K, Lausen B, Alldridge L, Leng L, Bucala R, Fernandez N, Metodiev MV. Author information: (1)The Helen Rollason Research Laboratory, Anglia Ruskin University, Chelmsford, Essex, CM1 1SQ, United Kingdom. Triple-negative breast cancer is difficult to treat because of the lack of rationale-based therapies. There are no established markers and targets that can be used for stratification of patients and targeted therapy. Here we report the identification of novel molecular features, which appear to augment metastasis of triple negative breast tumors. We found that triple-negative breast tumors can be segregated into 2 phenotypes based on their genome-wide protein abundance profiles. The first is characterized by high expression of Stat1, Mx1, and CD74. Seven out of 9 tumors from this group had invaded at least 2 lymph nodes while only 1 out of 10 tumors in group 2 was lymph node positive. In vitro experiments showed that the interferon-induced increase in Stat1 abundance correlates with increased migration and invasion in cultured cells. When CD74 was overexpressed, it increased cell adhesion on matrigel. This effect was accompanied with a marked increase in the membrane expression of beta-catenin, MUC18, plexins, integrins, and other proteins involved in cell adhesion and cancer metastasis. Taken together, our results show that Stat1/CD74 positive triple-negative tumors are more aggressive and suggest an approach for development of better diagnostics and more targeted therapies for triple negative breast cancer. This article is part of a Special Issue entitled: Proteomics: The clinical link. Copyright © 2011 Elsevier B.V. All rights reserved. DOI: 10.1016/j.jprot.2011.11.033 PMID: 22178447 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23436753
1. Proteomics Clin Appl. 2013 Apr;7(3-4):283-91. doi: 10.1002/prca.201200048. Epub 2013 Mar 6. Shotgun proteomics of archival triple-negative breast cancer samples. Gámez-Pozo A(1), Ferrer NI, Ciruelos E, López-Vacas R, Martínez FG, Espinosa E, Vara JÁ. Author information: (1)Laboratorio de Oncología y Patología Molecular, Instituto de Genética Médica y Molecular-INGEMM, Instituto de Investigación Hospital Universitario La Paz-IdiPAZ, Madrid, Spain. PURPOSE: Triple-negative breast cancer (TNBC) accounts for 15-20% of all breast cancers, and has a worse prognosis compared with hormone receptor-positive disease. Its unfavorable outcome and the lack of hormonal receptors determine the use of adjuvant chemotherapy as part of the standard treatment for these tumors, although several studies have documented that the current standard combination chemotherapy is suboptimal. Therefore, a new functional taxonomy of breast cancer and new targets for therapeutic development are urgently needed. EXPERIMENTAL DESIGN: In this study, we have analyzed the proteome of TNBC applying a high-throughput proteomics approach to routinely archived formalin-fixed, paraffin-embedded tumor tissues. RESULTS: We have been able to identify and quantify more than 1000 protein groups. Some of these proteins are of outstanding interest in the biology and clinical management of this disease, such as CD44 and PARP1. Moreover, we have characterized some signaling pathways that could be related to TNBC genesis and development. CONCLUSION AND CLINICAL RELEVANCE: Our results open up new avenues for the use of proteomics technologies in clinically relevant studies using archival samples. Shotgun LC-MS/MS studies could serve to discover new biomarkers and may provide clues to the genesis of TNBC and underlying molecular alterations. © 2013 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim. DOI: 10.1002/prca.201200048 PMID: 23436753 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22692575
1. Mol Med. 2012 Sep 25;18(1):1109-21. doi: 10.2119/molmed.2012.00091. Functional heterogeneity within the CD44 high human breast cancer stem cell-like compartment reveals a gene signature predictive of distant metastasis. Leth-Larsen R(1), Terp MG, Christensen AG, Elias D, Kühlwein T, Jensen ON, Petersen OW, Ditzel HJ. Author information: (1)Department of Cancer and Inflammation Research, Institute of Molecular Medicine, University of Southern Denmark, Odense, Denmark. The CD44(hi) compartment in human breast cancer is enriched in tumor-initiating cells; however, the functional heterogeneity within this subpopulation remains poorly defined. We used a triple-negative breast cancer cell line with a known bilineage phenotype to isolate and clone CD44(hi) single cells that exhibited mesenchymal/basal B and luminal/basal A features, respectively. Herein, we demonstrate in this and other triple-negative breast cancer cell lines that, rather than CD44(hi)/CD24(-) mesenchymal-like basal B cells, the CD44(hi)/CD24(lo) epithelioid basal A cells retained classic cancer stem cell features, such as tumor-initiating capacity in vivo, mammosphere formation and resistance to standard chemotherapy. These results complement previous findings using oncogene-transformed normal mammary cells showing that only cell clones with a mesenchymal phenotype exhibit cancer stem cell features. Further, we performed comparative quantitative proteomic and gene array analyses of these cells and identified potential novel markers of breast cancer cells with tumor-initiating features, such as lipolysis-stimulated lipoprotein receptor (LSR), RAB25, S100A14 and mucin 1 (MUC1), as well as a novel 31-gene signature capable of predicting distant metastasis in cohorts of estrogen receptor-negative human breast cancers. These findings strongly favor functional heterogeneity in the breast cancer cell compartment and hold promise for further refinements of prognostic marker profiling. Our work confirms that, in addition to cancer stem cells with mesenchymal-like morphology, those tumor-initiating cells with epithelial-like morphology should also be the focus of drug development. DOI: 10.2119/molmed.2012.00091 PMCID: PMC3474436 PMID: 22692575 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/21630460
1. Proteomics. 2011 Jul;11(13):2683-92. doi: 10.1002/pmic.201000801. Epub 2011 May 31. Quantitative chemical proteomics in small-scale culture of phorbol ester stimulated basal breast cancer cells. Dolai S(1), Xu Q, Liu F, Molloy MP. Author information: (1)Department of Chemistry and Biomolecular Sciences, Faculty of Science, Macquarie University, Sydney, Australia. Basal-like breast cancers are commonly negative for expression of estrogen and progesterone receptors and HER-2 (triple-negative breast cancer), which makes this subtype of breast cancers more aggressive and less responsive to standard treatment. We have applied a small-scale chemical proteomics method using bisindolylmaleimide (Bis) class of protein kinase C inhibitors to study the Bis-binding proteome in a cell culture model of basal breast carcinoma (MDA-MB-231). Using MS, we identified 174 proteins captured by the Bis-probe in phorbol ester (PMA) stimulated cells. Gene ontology analysis broadly categorised these proteins as ATP binding (42%), GTP binding (6%) and having nucleoside-triphosphatase activity (21%). Of the 64 enzymes captured by the Bis-probe, the majority had either ATP and/or nucleotide binding functions. Two previously unreported Bis binding protein kinases, serine/arginine-rich protein-specific kinase 1 (SRPK1) and interferon-induced RNA-dependent protein kinase (PKR) were observed. We then incorporated SILAC for quantitation to examine the proteins that were differentially captured by the Bis-probe following 30 and 60 min PMA stimulation. This provided novel evidence for PMA regulation of the enzymes glyceraldehyde-3-phosphate dehydrogenase, nucleolar RNA helicase 2 and Heterogeneous nuclear ribonucleoprotein M. Copyright © 2011 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim. DOI: 10.1002/pmic.201000801 PMID: 21630460 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23185200
1. Arch Med Sci. 2012 Nov 9;8(5):886-91. doi: 10.5114/aoms.2012.31620. Epub 2012 Nov 7. Lipid and protein oxidation in female patients with chronic fatigue syndrome. Tomic S(1), Brkic S, Maric D, Mikic AN. Author information: (1)Clinic for Infectious Diseases, Clinical Center Vojvodina, Novi Sad, Serbia. INTRODUCTION: Chronic fatigue syndrome (CFS) is a widely recognized problem, characterized by prolonged, debilitating fatigue and a characteristic group of accompanying symptoms, that occurs four times more frequently in women than in men. The aim of the study was to determine the existence of oxidative stress and its possible consequences in female patients with CFS. MATERIAL AND METHODS: Twenty-four women aged 15-45 who fulfilled the diagnostic criteria for CFS with no comorbidities were recruited and were age matched to a control group of 19 healthy women. After conducting the routine laboratory tests, levels of the lipid oxidation product malondialdehyde (MDA) and protein oxidation protein carbonyl (CO) were determined. RESULTS: The CFS group had higher levels of triglycerides (p = 0.03), MDA (p = 0.03) and CO (p = 0.002) and lower levels of HDL cholesterol (p = 0.001) than the control group. There were no significant differences in the levels of total protein, total cholesterol or LDL cholesterol. CONCLUSIONS: The CFS group had an unfavorable lipid profile and signs of oxidative stress induced damage to lipids and proteins. These results might be indicative of early proatherogenic processes in this group of patients who are otherwise at low risk for atherosclerosis. Antioxidant treatment and life style changes are indicated for women with CFS, as well as closer observation in order to assess the degree of atherosclerosis. DOI: 10.5114/aoms.2012.31620 PMCID: PMC3506242 PMID: 23185200
http://www.ncbi.nlm.nih.gov/pubmed/20597947
1. Dermatol Ther. 2010 May-Jun;23(3):291-8. doi: 10.1111/j.1529-8019.2010.01325.x. Burning mouth syndrome. Torgerson RR(1). Author information: (1)Department of Dermatology, Mayo Clinic, Rochester, MN 55905, USA. torgerson.rochelle@mayo.edu Burning mouth syndrome (BMS) is a chronic condition characterized by burning of the oral mucosa, with or without dysgeusia and xerostomia, in the setting of no underlying systemic disease or identifiable abnormalities on physical examination or laboratory testing. BMS disproportionately affects postmenopausal women. The pathophysiology of the disease is unknown; no single treatment has proven universally successful. In light of these shortcomings, having a practical approach to the evaluation and management of patients with BMS can improve both patient quality of life and physician satisfaction. DOI: 10.1111/j.1529-8019.2010.01325.x PMID: 20597947 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/9844361
1. Mt Sinai J Med. 1998 Oct-Nov;65(5-6):343-7. Burning mouth syndrome. Miyamoto SA(1), Ziccardi VB. Author information: (1)Department of Oral and Maxillofacial Surgery, New Jersey Dental School, University of Medicine and Dentistry of New Jersey, Newark 07103-2400, USA. Complaint of a burning mouth is an increasingly common problem in the aging population. This has remained an enigma for the treating clinician, because visible pathologic lesions or processes are usually not evident. Local, systemic and environmental causes must be assessed to elicit the predisposing factors. Some suggestions for managing burning mouth syndrome are offered. PMID: 9844361 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/11603307
1. J Dent Hyg. 2001 Summer;75(3):245-52; quiz 252-3, 255. Burning mouth syndrome. Perno M. A critical component of the dental hygiene process of care is assessment of the oral and general health conditions of clients. Some clients present with burning and painful sensations in the oral cavity in the absence of any noticeable disease. This condition has been referred to as burning mouth syndrome (BMS), an often complicated condition. Various local, systemic, and psychological factors have been linked with BMS, but its etiology is not fully understood. Yet as many as one million people are affected by it in the United States, and it is an increasingly-common problem in the aging population. Middle-aged women, mostly postmenopausal, are diagnosed with symptoms seven times more frequently than men. Careful diagnosis and treatment are necessary to alleviate the symptoms of this condition. Referral to a physician is warranted in some cases. The purposes of this course are to review the etiologic factors and clinical implications related to this condition and to discuss appropriate dental hygiene interventions. Collaboration among the client, dental hygienist, dentist, and physician provides for interdisciplinary actions that can lead to palliation of symptoms and evaluation of the possible underlying factors contributing to the condition. PMID: 11603307 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/18305436
1. Med Oral Patol Oral Cir Bucal. 2008 Mar 1;13(3):E167-70. Drug-induced burning mouth syndrome: a new etiological diagnosis. Salort-Llorca C(1), Mínguez-Serra MP, Silvestre FJ. Author information: (1)Service of Pharmacy, Mútua de Terrassa Hospital, Barcelona, Spain. Burning mouth syndrome (BMS) is defined as a burning sensation of the oral mucosa, in the absence of specific oral lesions. The underlying etiology remains unclear. Peripheral alterations may be related to the density or reactive capacity of the oral mucosal membrane receptors - these being largely influenced by BMS-related risk factors such as stress, anxiety, the female gender, climacterium and advanced age. The present study compiles the cases of BMS induced by drugs reported in the literature, and attempts to draw a series of conclusions. A search was conducted in the PubMed database using the following key words: burning mouth syndrome, drug-induced, antihypertensive and chemically-induced. The search was carried out in April 2007. The literature yielded clinical cases in which oral burning sensation is described after the administration of drugs belonging to different therapeutic groups: antiretrovirals, antiseizure drugs, hormones and particularly antihypertensive medication. Curiously, among the different types of antihypertensive drugs, BMS was only associated with those compounds that act upon the angiotensin-renin system. PMID: 18305436 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/15388940
1. Acta Crystallogr D Biol Crystallogr. 2004 Oct;60(Pt 10):1883-7. doi: 10.1107/S0907444904018232. Epub 2004 Sep 23. Expression, purification, crystallization and preliminary structural characterization of the GTPase domain of human Rheb. Yu Y(1), Chang Y, Li S, Hu H, Huang Q, Ding J. Author information: (1)Key Laboratory of Proteomics, Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, 320 Yue-Yang Road, Shanghai 200031, People's Republic of China. Ras homologue enriched in brain (Rheb) represents a unique group of small GTPases and shares moderate sequence identity with the Ras/Rap subfamily. It acts downstream of nutrient signalling as the direct target of the tuberous sclerosis complex (TSC) and upstream of mTOR/S6K1/4EBP in the insulin-signalling pathway. The GTPase domain of human Rheb (hRheb) has been recombinantly expressed in Escherichia coli, purified and cocrystallized in complexes with GDP, GTP and GppNHp using the hanging-drop vapour-diffusion method. Crystals of the hRheb-GDP complex belong to space group P2(1)2(1)2(1), with unit-cell parameters a = 44.5, b = 52.3, c = 70.6 A. The hRheb-GppNHp complex crystallized in two crystal forms: one has the same space group and unit-cell parameters as the hRheb-GDP complex and the other belongs to space group C222(1), with unit-cell parameters a = 102.9, b = 99.2, c = 48.0 A. The hRheb-GTP complex also crystallized in two crystal forms: one belongs to space group C222(1), with unit-cell parameters a = 102.4, b = 98.3, c = 47.9 A, and the other belongs to space group P2(1), with unit-cell parameters a = 77.3, b = 47.9, c = 71.9 A, beta = 89.0 degrees. All these crystals diffract X-rays to better than 2.8 A resolution and at least one diffraction data set has been collected for each crystal form using an in-house R-AXIS IV++ diffractometer. Structural studies of hRheb in complexes with various substrates may provide insights into the recognition and specificity of substrate and the catalytic mechanism of mammalian Rhebs and shed light on the biological functions of Rhebs in the mTOR signalling pathway. DOI: 10.1107/S0907444904018232 PMID: 15388940 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/20457704
1. Arch Dis Child. 2010 May;95(5):391-2. doi: 10.1136/adc.2009.159210. Sirolimus and tuberous sclerosis-associated renal angiomyolipomas. Krischock L(1), Beach R, Taylor J. Author information: (1)Department of Paediatric Nephrology, Evelina Children's Hospital, Guy's and St Thomas' Hospitals NHS Trust, London, UK. Tuberous sclerosis, caused by germline mutations in the TSC1 or TSC2 genes, is associated with aberrant upregulation of the mammalian target of rapamycin (mTOR) signalling pathway, resulting in growth of tumours, including renal angiomyolipomas (AMLs). AMLs may cause hypertension, renal failure and spontaneous life-threatening haemorrhage. Previously, invasive interventions were required to treat AMLs. More recently, mTOR inhibitors have been used as molecularly targeted treatment to treat AMLs. We present here the case of a paediatric patient with TSC in whom sirolimus has been used successfully to halt growth of renal AMLs. DOI: 10.1136/adc.2009.159210 PMID: 20457704 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/12172555
1. Nat Cell Biol. 2002 Sep;4(9):699-704. doi: 10.1038/ncb847. Tsc tumour suppressor proteins antagonize amino-acid-TOR signalling. Gao X(1), Zhang Y, Arrazola P, Hino O, Kobayashi T, Yeung RS, Ru B, Pan D. Author information: (1)Department of Physiology, University of Texas Southwestern Medical Center at Dallas, 5323 Harry Hines Blvd., Dallas, TX 75390-9040, USA. Comment in Nat Cell Biol. 2002 Sep;4(9):E214-6. doi: 10.1038/ncb0902-e214. Target of Rapamycin (TOR) mediates a signalling pathway that couples amino acid availability to S6 kinase (S6K) activation, translational initiation and cell growth. Here, we show that tuberous sclerosis 1 (Tsc1) and Tsc2, tumour suppressors that are responsible for the tuberous sclerosis syndrome, antagonize this amino acid-TOR signalling pathway. We show that Tsc1 and Tsc2 can physically associate with TOR and function upstream of TOR genetically. In Drosophila melanogaster and mammalian cells, loss of Tsc1 and Tsc2 results in a TOR-dependent increase of S6K activity. Furthermore, although S6K is normally inactivated in animal cells in response to amino acid starvation, loss of Tsc1-Tsc2 renders cells resistant to amino acid starvation. We propose that the Tsc1-Tsc2 complex antagonizes the TOR-mediated response to amino acid availability. Our studies identify Tsc1 and Tsc2 as regulators of the amino acid-TOR pathway and provide a new paradigm for how proteins involved in nutrient sensing function as tumour suppressors. DOI: 10.1038/ncb847 PMID: 12172555 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23159330
1. Neuropharmacology. 2013 Apr;67:1-7. doi: 10.1016/j.neuropharm.2012.11.003. Epub 2012 Nov 14. Behavioural and EEG effects of chronic rapamycin treatment in a mouse model of tuberous sclerosis complex. Cambiaghi M(1), Cursi M, Magri L, Castoldi V, Comi G, Minicucci F, Galli R, Leocani L. Author information: (1)San Raffaele Scientific Institute, Milan, Italy. cambiaghi.marco@hsr.it Tuberous Sclerosis Complex (TSC) is a multisystem genetic disorder caused by mutation in either Tsc1 or Tsc2 genes that leads to the hyper activation of the mTOR pathway, a key signalling pathway for synaptic plasticity. TSC is characterized by benign tumors arising in different organs and severe neuropsychiatric symptoms, such as epilepsy, intellectual disability, autism, anxiety and depressive behaviour. Rapamycin is a potent inhibitor of mTOR and its efficacy in treating epilepsy and neurological symptoms remains elusive. In a mouse model in which Tsc1 has been deleted in embryonic telencephalic neural stem cells, we analyzed anxiety- and depression-like behaviour by elevated-plus maze (EPM), open-field test (OFT), forced-swim test (FST) and tail-suspension test (TST), after chronic administration of rapamycin. In addition, spectral analysis of background EEG was performed. Rapamycin-treated mutant mice displayed a reduction in anxiety- and depression-like phenotype, as shown by the EPM/OFT and FST, respectively. These results were inline with EEG power spectra outcomes. The same effects of rapamycin were observed in wild-type mice. Notably, in heterozygous animals we did not observe any EEG and/or behavioural variation after rapamycin treatment. Together these results suggest that both TSC1 deletion and chronic rapamycin treatment might have a role in modulating behaviour and brain activity, and point out to the potential usefulness of background EEG analysis in tracking brain dysfunction in parallel with behavioural testing. Copyright © 2012 Elsevier Ltd. All rights reserved. DOI: 10.1016/j.neuropharm.2012.11.003 PMID: 23159330 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/19506736
1. Curr Genomics. 2008 Nov;9(7):475-87. doi: 10.2174/138920208786241243. Genetics and molecular biology of tuberous sclerosis complex. Napolioni V(1), Curatolo P. Author information: (1)Laboratory of Human Genetics, Department of Molecular, Cellular and Animal Biology, University of Camerino, Camerino, Italy. Tuberous Sclerosis Complex is a multisystem disorder exhibiting a wide range of manifestations characterized by tumour-like lesions called hamartomas in the brain, skin, eyes, heart, lungs and kidneys. Tuberous Sclerosis Complex is genetically determined with an autosomal dominant inheritance and is caused by inactivating mutations in either the TSC1 or TSC2 genes. TSC1/2 genes play a fundamental role in the regulation of phosphoinositide 3-kinase (PI3K) signalling pathway, inhibiting the mammalian target of rapamycin (mTOR) through activation of the GTPase activity of Rheb. Mutations in TSC1/2 genes impair the inhibitory function of the hamartin/tuberin complex, leading to phosphorylation of the downstream effectors of mTOR, p70 S6 kinase (S6K), ribosomal protein S6 and the elongation factor binding protein 4E-BP1, resulting in uncontrolled cell growth and tumourigenesis.Despite recent promising genetic, diagnostic, and therapeutic advances in Tuberous Sclerosis Complex, continuing research in all aspects of this complex disease will be pivotal to decrease its associated morbidity and mortality. In this review we will discuss and analyse all the important findings in the molecular pathogenesis of Tuberous Sclerosis Complex, focusing on genetics and the molecular mechanisms that define this multisystemic disorder. DOI: 10.2174/138920208786241243 PMCID: PMC2691673 PMID: 19506736
http://www.ncbi.nlm.nih.gov/pubmed/12556239
1. Ann Hum Genet. 2003 Jan;67(Pt 1):87-96. doi: 10.1046/j.1469-1809.2003.00012.x. Tuberous sclerosis: from tubers to mTOR. Kwiatkowski DJ(1). Author information: (1)Hematology Division, Department of Medicine, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA. dk@rics.bwh.harvard.edu Tuberous sclerosis (TSC) is an autosomal dominant hamartoma syndrome whose causative genes (TSC1 and TSC2) were identified 5 and 9 years ago respectively. Their encoded proteins are large, and apart from a strong binding interaction with each other, relatively little was known about their biochemical function. Recent studies in Drosophila have pinpointed a critical function for the DrosophilaTSC1/TSC2 homologues in the regulation of cell size. Epistasis experiments and a variety of biochemical studies that followed have indicated a critical function for these proteins in the highly conserved PI-3-kinase-Akt-mTOR signalling pathway. DOI: 10.1046/j.1469-1809.2003.00012.x PMID: 12556239 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/12937031
1. J Appl Physiol (1985). 2003 Dec;95(6):2355-60. doi: 10.1152/japplphysiol.00651.2003. Epub 2003 Aug 22. Effect of exercise intensity on the postexercise sweating threshold. Kenny GP(1), Periard J, Journeay WS, Sigal RJ, Reardon FD. Author information: (1)Human Performance and Environmental Physiology Research Laboratory, School of Human Kinetics, Faculty of Health Sciences, University of Ottawa, Ottawa, Ontario, Canada K1N 6N5. gkenny@uottawa.ca The hypothesis that the magnitude of the postexercise onset threshold for sweating is increased by the intensity of exercise was tested in eight subjects. Esophageal temperature was monitored as an index of core temperature while sweat rate was measured by using a ventilated capsule placed on the upper back. Subjects remained seated resting for 15 min (no exercise) or performed 15 min of treadmill running at either 55, 70, or 85% of peak oxygen consumption (V(o2 peak)) followed by a 20-min seated recovery. Subjects then donned a liquid-conditioned suit used to regulate mean skin temperature. The suit was first perfused with 20 degrees C water to control and stabilize skin and core temperature before whole body heating. Subsequently, the skin was heated ( approximately 4.0 degrees C/h) until sweating occurred. Exercise resulted in an increase in the onset threshold for sweating of 0.11 +/- 0.02, 0.23 +/- 0.01, and 0.33 +/- 0.02 degrees C above that measured for the no-exercise resting values (P < 0.05) for the 55, 70, and 85% of V(o2 peak) exercise conditions, respectively. We did note that there was a greater postexercise hypotension as a function of exercise intensity as measured at the end of the 20-min exercise recovery. Thus it is plausible that the increase in postexercise threshold may be related to postexercise hypotension. It is concluded that the sweating response during upright recovery is significantly modified by exercise intensity and may likely be influenced by the nonthermal baroreceptor reflex adjustments postexercise. DOI: 10.1152/japplphysiol.00651.2003 PMID: 12937031 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/2703281
1. Int J Sports Med. 1989 Feb;10(1):25-9. doi: 10.1055/s-2007-1024868. Effect of active warming-up on thermoregulatory, circulatory, and metabolic responses to incremental exercise in endurance-trained athletes. Chwalbińska-Moneta J(1), Hänninen O. Author information: (1)Department of Applied Physiology, Medical Research Center, Polish Academy of Sciences, Warsaw. The influence of 10 min warming-up at 40% VO2 max on thermal, circulatory, and metabolic responses to an incremental exercise to exhaustion as well as on the anaerobic threshold at the blood lactate level of 4 mmol.l-1 (AT) and the individual anaerobic threshold (IAT) was investigated in eight cross-country skiers. During exercise preceded by warming-up, the mean skin temperature (T sk) and external auditory canal temperature (Tac) did not change significantly in contrast to exercise without warming-up, producing a rise in both T sk and Tac (by approx. 1.2 degrees C and 1.1 degrees C, respectively). Warming-up did not alter the course of the rectal temperature changes during exercise. With warming-up skin humidity rose immediately after the beginning of exercise, whereas the onset of sweating without warming-up appeared much later at higher work intensities. Warming-up did not change the circulatory and ventilatory responses to incremental exercise and the oxygen uptake (VO2) either at submaximal or maximal work loads. With warming-up a significant increase was found in the threshold work load both at the AT and the IAT. The data demonstrated that warming-up has an advantageous effect on the efficiency of thermoregulation in endurance-trained athletes producing an early sweating response to the incremental exercise that results in attenuation of hyperthermia. An increase in the anaerobic threshold during incremental exercise preceded by warming-up may indicate an enhancement of the endurance capacity subsequent to warming-up. DOI: 10.1055/s-2007-1024868 PMID: 2703281 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/21623345
1. EMBO J. 2011 May 27;30(13):2596-609. doi: 10.1038/emboj.2011.166. The DNA-binding domain of the Chd1 chromatin-remodelling enzyme contains SANT and SLIDE domains. Ryan DP(1), Sundaramoorthy R, Martin D, Singh V, Owen-Hughes T. Author information: (1)Wellcome Trust Centre for Gene Regulation and Expression, College of Life Sciences, University of Dundee, Dundee, UK. The ATP-dependent chromatin-remodelling enzyme Chd1 is a 168-kDa protein consisting of a double chromodomain, Snf2-related ATPase domain, and a C-terminal DNA-binding domain. Here, we show the DNA-binding domain is required for Saccharomyces cerevisiae Chd1 to bind and remodel nucleosomes. The crystal structure of this domain reveals the presence of structural homology to SANT and SLIDE domains previously identified in ISWI remodelling enzymes. The presence of these domains in ISWI and Chd1 chromatin-remodelling enzymes may provide a means of efficiently harnessing the action of the Snf2-related ATPase domain for the purpose of nucleosome spacing and provide an explanation for partial redundancy between these proteins. Site directed mutagenesis was used to identify residues important for DNA binding and generate a model describing the interaction of this domain with DNA. Through inclusion of Chd1 sequences in homology searches SLIDE domains were identified in CHD6-9 proteins. Point mutations to conserved amino acids within the human CHD7 SLIDE domain have been identified in patients with CHARGE syndrome. DOI: 10.1038/emboj.2011.166 PMCID: PMC3155300 PMID: 21623345 [Indexed for MEDLINE] Conflict of interest statement: The authors declare that they have no conflict of interest.
http://www.ncbi.nlm.nih.gov/pubmed/24173008
1. Am J Health Syst Pharm. 2013 Nov 15;70(22):1984-94. doi: 10.2146/ajhp130199. A review of tuberculosis: Focus on bedaquiline. Chan B(1), Khadem TM, Brown J. Author information: (1)Bonnie Chan, Pharm.D., is Assistant Professor of Pharmacy, School of Pharmacy, Philadelphia College of Osteopathic Medicine, Suwanee, GA; at the time of writing she was Postgraduate Year 2 Infectious Diseases Pharmacy Resident, Department of Pharmacy, University of Rochester Medical Center (URMC), Rochester, NY. Tina M. Khadem, Pharm.D., is Postdoctoral Research Fellow, Department of Pharmacy Practice, Wegmans School of Pharmacy, St. John Fisher College, Rochester, and Postdoctoral Research Fellow, Department of Pharmacy, URMC. Jack Brown, Pharm.D., M.S., is Associate Professor and Chair, Department of Pharmacy Practice and Administration, Wegmans School of Pharmacy, St. John Fisher College, and Adjunct Research Assistant Professor, Department of Social and Preventative Medicine, URMC. PURPOSE: The history and prevalence of tuberculosis and the role of bedaquiline in multidrug-resistant (MDR) tuberculosis are reviewed. SUMMARY: Tuberculosis continues to cause significant morbidity and mortality worldwide. Increasing rates of drug-resistant tuberculosis are a significant concern and pose serious implications for current and future treatment of the disease. In December 2012, the Food and Drug Administration approved bedaquiline as part of the treatment regimen for pulmonary MDR tuberculosis. Bedaquiline's unique mechanism of action presents an alternative approach to current antimycobacterial killing. By directly inhibiting adenosine triphosphate (ATP) synthase, bedaquiline is effective against both replicating and dormant mycobacteria. Pulmonary cavitary lesions can contain heterogeneous populations. This potential mix of semireplicating and hypometabolic mycobacteria is more difficult to eliminate with conventional antitubercular drugs, thus increasing the risk of resistance. No in vitro cross-resistance between bedaquiline and currently available antitubercular agents has been observed thus far. Because bedaquiline targets a completely different enzyme, cross-resistance with other conventional agents remains unlikely. Enhanced sterilizing capacity via synergistic depletion of ATP further exhibits the promising potential of bedaquiline with pyrazinamide. A course of bedaquiline requires 24 weeks of therapy in combination with other antitubercular drugs. CONCLUSION: The approval of bedaquiline represents a major milestone in MDR tuberculosis therapy. Bedaquiline should be considered in patients who have not responded to a regimen containing four second-line drugs and pyrazinamide and patients with documented evidence of MDR tuberculosis resistant to fluoroquinolones. The exact role of bedaquiline cannot be determined until further efficacy and safety data are obtained through ongoing Phase III trials. DOI: 10.2146/ajhp130199 PMID: 24173008 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/19031760
1. Zhonghua Liu Xing Bing Xue Za Zhi. 2008 Jul;29(7):683-4. [Molecular epidemiological study on oxidative DNA damage among Hasake ethnic migrants in Shenzhen]. [Article in Chinese] Ke YB(1), Cheng JQ, Shuai ZH, Zhang RL, Jiang GF, Tan CR, Zhang ZZ. Author information: (1)Department of Genetic Toxicology, Shenzhen Center for Disease Control and Prevention, Shenzhen 518020, China. OBJECTIVE: To explore the relationship of migration and oxidative DNA damage by comparative study of oxidative DNA damage effects on people with different years of migration among Xinjiang Hasake ethnicity in Shenzhen. METHODS: Sixty Hasake residents in Shenzhen were selected, and were divided into three groups (n=20) according to the years of migration. Major changes of their life style were investigated. 8-hydroxy-2'-deoxyguanosine (8-OH-dG) levels in urine were analyzed, and comet assay of peripheral blood lymphocytes conducted. RESULTS: When comparing with the group having a shorter than 1 year of stay,a significant decrease of oliveira tail moment and tail/head length in comet assay in the >3 years group (P < 0.05) was observed 8-OH-dG level decreased significantly in 1-3 years group (P < 0.05) and >3 years group (P < 0.01). CONCLUSION: Our results suggested that life style changes which related to migration might reduce DNA damage in Hasake nationalities. PMID: 19031760 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23105513
1. Indian J Clin Biochem. 2005 Jan;20(1):145-9. doi: 10.1007/BF02893061. Oxidative stress in metabolic syndrome. Sharma P(1), Mishra S, Ajmera P, Mathur S. Author information: (1)Department of Biochemistry, S.M.S. Medical College, 302 004 Jaipur, India. As antioxidants play a protective role in the pathophysiology of diabetes and cardiovascular diseases, understanding the physiological status of antioxidant concentration among people at high risk for developing these conditions, such as Metabolic Syndrome, is of interest. In present study out of 187 first degree non-diabetic relatives and 192 non-diabetic spouses, 33.1% and 19.7% were found to have metabolic syndrome respectively. Subjects with metabolic syndrome (≥3 risk factors) had poor antioxidants status as reflected by significantly low levels of vitamin A, C & E and significantly increased (p<0.01) oxidative stress as compared to those without metabolic syndrome. At the same time serum insulin levels and insulin resistance were found to be significantly high (p<0.001) in metabolic syndrome. A strong positive correlation (r=0.946; p<0.001) between oxidative stress and insulin resistance was observed in metabolic syndrome. Low levels of antioxidants and increased oxidative stress with insulin resistance in metabolic syndrome suggests that besides therapeutic life style changes (TLC) as suggested in ATP III guidelines inclusion of antioxidant vitamins, fruits and vegetable could be beneficial to ward off the consequences of metabolic syndrome. DOI: 10.1007/BF02893061 PMCID: PMC3454143 PMID: 23105513
http://www.ncbi.nlm.nih.gov/pubmed/7588694
1. Eur J Appl Physiol Occup Physiol. 1995;71(2-3):235-9. doi: 10.1007/BF00854984. Effect of aerobic capacity on sweat rate and fluid intake during outdoor exercise in the heat. Yoshida T(1), Nakai S, Yorimoto A, Kawabata T, Morimoto T. Author information: (1)Department of Physiology, Kyoto Prefectural University of Medicine, Japan. We measured the aerobic capacity, sweat rate and fluid intake of trained athletes during outdoor exercise and examined the relationship between aerobic capacity and thermoregulatory responses at high ambient temperatures. The maximal aerobic capacity (VO2max) of the subjects, nine male baseball players of college age, was determined by maximal exercise tests on a cycle ergometer. The subjects practised baseball regularly without drinking fluids from 1330 to 1530 hours. After 30 min rest, they played a baseball game with free access to a sports drink at 15 degrees C from 1600 to 1830 hours. At a mean ambient temperature of 36.7 (SEM 0.2) degree C, the mean percentage of body mass loss (delta mb) and increase of oral temperature (delta To) from 1330 to 1530 hours was 3.47 (SEM 0.12)% and 0.81 (SEM 0.14) degree C, respectively. The sweat loss from 1330 to 1830 hours was 56.53 (SEM 1.56)ml.kg-1 of body mass (mb) while the mean fluid consumption was 44.78 (SEM 2.39)ml.kg-1 of mb, with recovery of 76.08 (SEM 2.81)% of sweat loss. The VO2max was significantly inversely correlated with delta mb, fluid intake and rehydration amount, but showed no correlation with delta To. These results would suggest that at a given exercise intensity in subjects with a higher aerobic capacity body temperature is maintained with a lower sweating rate than that in subjects with a lower aerobic capacity. DOI: 10.1007/BF00854984 PMID: 7588694 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/20005186
1. Mol Oncol. 2010 Feb;4(1):65-89. doi: 10.1016/j.molonc.2009.11.003. Epub 2009 Nov 23. Up-regulated proteins in the fluid bathing the tumour cell microenvironment as potential serological markers for early detection of cancer of the breast. Gromov P(1), Gromova I, Bunkenborg J, Cabezon T, Moreira JM, Timmermans-Wielenga V, Roepstorff P, Rank F, Celis JE. Author information: (1)Institute of Cancer Biology, Danish Cancer Society, DK-2100 Copenhagen, Denmark. psg@cancer.dk <psg@cancer.dk> Breast cancer is by far the most common diagnosed form of cancer and the leading cause of cancer death in women today. Clinically useful biomarkers for early detection of breast cancer could lead to a significant reduction in mortality. Here we describe a detailed analysis using gel-based proteomics in combination with mass spectrometry and immunohistochemistry (IHC) of the tumour interstitial fluids (TIF) and normal interstitial fluids (NIF) collected from 69 prospective breast cancer patients. The goal of this study was to identify abundant cancer up-regulated proteins that are externalised by cells in the tumour microenvironment of most if not all these lesions. To this end, we applied a phased biomarker discovery research strategy to the analysis of these samples rather than comparing all samples among each other, with inherent inter and intra-sample variability problems. To this end, we chose to use samples derived from a single tumour/benign tissue pair (patient 46, triple negative tumour), for which we had well-matched samples in terms of epithelial cell numbers, to generate the initial dataset. In this first phase we found 110 proteins that were up-regulated by a factor of 2 or more in the TIF, some of which were confirmed by IHC. In the second phase, we carried out a systematic computer assisted analysis of the 2D gels of the remaining 68 TIF samples in order to identify TIF 46 up-regulated proteins that were deregulated in 90% or more of all the available TIFs, thus representing common breast cancer markers. This second phase singled out a set of 26 breast cancer markers, most of which were also identified by a complementary analysis using LC-MS/MS. The expression of calreticulin, cellular retinoic acid-binding protein II, chloride intracellular channel protein 1, EF-1-beta, galectin 1, peroxiredoxin-2, platelet-derived endothelial cell growth factor, protein disulfide isomerase and ubiquitin carboxyl-terminal hydrolase 5 were further validated using a tissue microarray containing 70 malignant breast carcinomas of various grades of atypia. A significant number of these proteins have already been detected in the blood/plasma/secretome by others. The next steps, which include biomarker prioritization based on the hierarchal evaluation of these markers, antibody and antigen development, assay development, analytical validation, and preliminary testing in the blood of healthy and breast cancer patients, are discussed. DOI: 10.1016/j.molonc.2009.11.003 PMCID: PMC5527961 PMID: 20005186 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/9694410
1. Int J Sports Med. 1998 Jun;19 Suppl 2:S103-5. doi: 10.1055/s-2007-971969. Effects of training, environment, and host factors on the sweating response to exercise. Armstrong LE(1), Maresh CM. Author information: (1)University of Connecticut, Dept. of Physiology & Neurobiology, Storrs 06269-1110, USA. Armstron@uconnvm.uconn.edu Because metabolic heat production is proportional to the amount of work performed, the differences in core body temperature (Tcore) of humans exercising at similar absolute exercise intensities are due to differences in their efficiency of heat dissipation. The purpose of this paper is to delineate the effects of training status, heat acclimation, environmental conditions and host factors on the sweating response to exercise. These factors are reviewed in light of their effects on the biophysical enhancement or suppression of sweating, and modifications of the relationship between local sweat rate and Tcore (degrees C). Athletes are advised to optimize those factors that enhance, and eliminate those factors that diminish, the onset and responsiveness of sweating. DOI: 10.1055/s-2007-971969 PMID: 9694410 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/21669114
1. Neurol Res. 2011 Jun;33(5):467-72. doi: 10.1179/016164111X13007856083963. Chronic Helicobacter pylori infection and ischemic stroke subtypes. Yang X(1), Gao Y, Zhao X, Tang Y, Su Y. Author information: (1)Department of Neurology of Aerospace Central Hospital (Aerospace Clinical Medical College Affiliated to Peking University), Beijing 100049, China. OBJECTIVE: Chronic infection by Helicobacter pylori is regarded as an etiological factor for vascular diseases. However, there are conflicting results on the relevance of chronic infection by Helicobacter pylori as a risk factor for ischemic stroke. The aim of our study was to investigate the association between Helicobacter pylori infection and ischemic stroke subtypes in Chinese. METHOD: A total of 150 patients with ischemic stroke were enrolled in the patient group. Analyses were stratified for etiologic stroke subtypes according to 2007 modified Trial of Org 10172 in Acute Stroke Treatment criteria: 119 patients with atherothrombosis, 15 patients with cardioembolism, and 12 patients with small artery disease. One hundred and thirty-one control subjects without clinical and instrumental evidence of atherosclerotic diseases were randomly selected from health check-up center. The potential risk factors for Helicobacter pylori infection and traditional risk factors for ischemic stroke of all subjects were analyzed. The serum specific antibody IgG of Helicobacter pylori was detected by enzyme-linked immunosorbent assay. Conditional logistic regression was used to analyze the data. RESULTS: The Helicobacter pylori/IgG-positive rate in the patient group was higher than that in the healthy control group, but the difference was not statistically significant [67.3% versus 61.8%; odds ratio (OR) = 1.272; P = 0.336]. This result remained non-significant after adjustment for other established risk factors [OR = 1.222; 95% confidence interval (CI): 0.688-2.171; P = 0.494]. Subgroup analysis using univariate and multivariate analyses yielded similar results in all etiologic stroke subtypes (univariate analysis, atherothrombosis: OR = 1.368, 95%CI: 0.810-2.311, P = 0.241; cardioembolism: OR = 0.926, 95%CI:0.311-2.758, P = 0.890; small artery disease: OR = 1.852, 95%CI: 0.478-7.167, P = 0.366; multivariate analysis, atherothrombosis: OR = 1.385, 95%CI: 0.726-2.639, P = 0.323; cardioembolism: OR = 0.832, 95%CI: 0.236-2.932, P = 0.775; small artery disease: OR = 1.836, 95%CI: 0.396-8.503, P = 0.437). CONCLUSIONS: This case-control study does not reveal any strong association between chronic Helicobacter pylori infection and ischemic stroke. Large case-control prospective studies are required for further investigation of the potential association between Helicobacter pylori infection and ischemic stroke risk, particularly in certain subgroups. DOI: 10.1179/016164111X13007856083963 PMID: 21669114 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/15694938
1. Atherosclerosis. 2005 Feb;178(2):303-9. doi: 10.1016/j.atherosclerosis.2004.08.025. Association between chronic Helicobacter pylori infection and acute ischemic stroke: Fukuoka Harasanshin Atherosclerosis Trial (FHAT). Sawayama Y(1), Ariyama I, Hamada M, Otaguro S, Machi T, Taira Y, Hayashi J. Author information: (1)Division of General Medicine, Harasanshin General Hospital 1-8, Taihaku-cho, Hakata-ku, Fukuoka 812-0033, Japan. genmedpr@hrasanshin.or.jp Helicobacter pylori (H. pylori) have been associated both epidemiologically and pathogenetically with coronary atherosclerosis, but data on the relationship between chronic H. pylori infection and stroke are lacking. Therefore, we investigated the relationship between H. pylori infection and acute ischemic stroke in 62 patients with their first stroke and 143 controls. The stroke patients were all admitted to Harasanshin General Hospital (Fukuoka, Japan) and the controls were asymptomatic age-matched outpatients with hyperlipidemia who did not have cardiac disease or infections. All patients underwent cranial CT scanning and/or brain magnetic resonance imaging, duplex ultrasonography of the extracranial carotid arteries, and transthoracic echocardiography. H. pylori infection was diagnosed by detection of anti-H. pylori IgG antibodies, the 13C-urea breath test, and histology. Conditional logistic regression analysis was performed to analyze the data. The 62 stroke patients and 143 controls were aged from 41 to 92 years. Chronic H. pylori infection was associated with a higher risk of stroke due to small artery occlusion (odds ratio: 9.68; 95% CI: 3.56-33.08, P <0.001) and a lower risk of cardioembolic stroke (odds ratio: 0.27; 95% CI: 0.03-1.53). Chronic H. pylori infection still showed an overall association with ischemic stroke (odds ratio for all subtypes combined: 2.57; 95% CI: 1.09-6.08) after adjusting for major cardiovascular risk factors. These results suggest that chronic H. pylori infection may be a triggering factor that increases the risk of acute ischemic stroke. DOI: 10.1016/j.atherosclerosis.2004.08.025 PMID: 15694938 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22713083
1. J Dig Dis. 2012 Jul;13(7):342-9. doi: 10.1111/j.1751-2980.2012.00599.x. Extragastrointestinal manifestations of Helicobacter pylori infection: facts or myth? A critical review. Tan HJ(1), Goh KL. Author information: (1)Department of Gastroenterology, Sunway Medical Centre, Selangor Department of Gastroenterology, University of Malaya, Kuala Lumpur, Malaysia. hucktan@hotmail.com Helicobacter pylori (H. pylori) infection is reported to be associated with many extragastrointestinal manifestations, such as hematological diseases [idiopathic thrombocytopenic purpura (ITP) and unexplained iron deficiency anemia (IDA)], cardiovascular diseases (ischemic heart diseases), neurological disorders (stroke, Parkinson's disease, Alzheimer's disease), obesity and skin disorders. Among these, the best evidence so far is in ITP and unexplained IDA, with high-quality studies showing the improvement of IDA and ITP after H. pylori eradication. The evidence of its association with coronary artery disease is weak and many of the results may be erroneous. The role of H. pylori infection in affecting serum leptin and ghrelin levels has attracted a lot of attention recently and available data to date have been conflicting. There have also been many uncontrolled, small sample studies suggesting an association between H. pylori infection and neurological disorders or chronic urticaria. However, more studies are required to clarify such proposed causal links. © 2012 The Authors. Journal of Digestive Diseases © 2012 Chinese Medical Association Shanghai Branch, Chinese Society of Gastroenterology, Renji Hospital Affiliated to Shanghai Jiaotong University School of Medicine and Blackwell Publishing Asia Pty Ltd. DOI: 10.1111/j.1751-2980.2012.00599.x PMID: 22713083 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/21060340
1. Nat Rev Neurol. 2010 Dec;6(12):681-94. doi: 10.1038/nrneurol.2010.163. Epub 2010 Nov 9. Common infections and the risk of stroke. Grau AJ(1), Urbanek C, Palm F. Author information: (1)Neurology Department, Klinikum Ludwigshafen am Rhein, Bremserstrasse 79, Ludwigshafen am Rhein, Germany. graua@klilu.de The occurrence of stroke in populations is incompletely explained by traditional vascular risk factors. Data from several case-control studies and one large study using case series methodology indicate that recent infection is a temporarily acting, independent trigger factor for ischemic stroke. Both bacterial and viral infections, particularly respiratory tract infections, contribute to this association. A causal role for infection in stroke is supported by a graded temporal relationship between these conditions, and by multiple pathophysiological pathways linking infection and inflammation, thrombosis, and stroke. Furthermore, observational studies suggest that influenza vaccination confers a preventive effect against stroke. Case-control and prospective studies indicate that chronic infections, such as periodontitis, chronic bronchitis and infection with Helicobacter pylori, Chlamydia pneumoniae or Cytomegalovirus, might increase stroke risk, although considerable variation exists in the results of these studies, and methodological issues regarding serological results remain unresolved. Increasing evidence indicates that the aggregate burden of chronic and/or past infections rather than any one single infectious disease is associated with the risk of stroke. Furthermore, genetic predispositions relating to infection susceptibility and the strength of the inflammatory response seem to co-determine this risk. Here, we summarize and analyze the evidence for common acute and chronic infectious diseases as stroke risk factors. DOI: 10.1038/nrneurol.2010.163 PMID: 21060340 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22012946
1. Pediatr Blood Cancer. 2012 Aug;59(2):246-53. doi: 10.1002/pbc.23357. Epub 2011 Oct 19. Initial testing of the investigational NEDD8-activating enzyme inhibitor MLN4924 by the pediatric preclinical testing program. Smith MA(1), Maris JM, Gorlick R, Kolb EA, Lock R, Carol H, Keir ST, Reynolds CP, Kang MH, Morton CL, Wu J, Smith PG, Yu J, Houghton PJ. Author information: (1)Cancer Therapy Evaluation Program, NCI, Bethesda, Maryland 20892, USA. smithm@ctep.nci.nih.gov Erratum in Pediatr Blood Cancer. 2012 Oct;59(4):772. BACKGROUND: MLN4924 is an investigational first-in-class small molecule inhibitor of NEDD8-activating enzyme (NAE). NAE is an essential component of the NEDD8 conjugation pathway, controlling the activity of a subset of ubiquitin-proteasome system (UPS) E3 ligases, multiprotein complexes that transfer ubiquitin molecules to substrate proteins. PROCEDURES: MLN4924 was tested against the PPTP in vitro panel using 96-hour exposure time at concentrations ranging from 1.0 nM to 10 µM. It was tested in vivo at a dose of 100 mg/kg [66 mg/kg for the acute lymphoblastic leukemia (ALL) xenografts] administered orally twice daily × 5 days. Treatment duration was 3 weeks. RESULTS: The median relative IC(50) for MLN4924 against the PPTP cell lines was 143 nM, (range: 15-678 nM) with that for the Ewing panel being significantly lower (31 nM). MLN4924 induced significant differences in EFS distribution compared to control in 20 of 34 (59%) evaluable solid tumor xenografts. MLN4924 induced intermediate activity (EFS T/C values >2) in 9 of the 33 evaluable xenografts (27%), including 4 of 4 glioblastoma xenografts, 2 of 3 Wilm's tumor xenografts, 2 of 5 rhabdomyosarcoma xenografts, and 1 of 4 neuroblastoma xenografts. For the ALL panel, 5 of 8 evaluable xenografts showed intermediate activity for the EFS T/C measure. MLN4924 did not induce objective responses in the PPTP solid tumor or ALL panels. CONCLUSIONS: MLN4924 showed potent activity in vitro and in vivo showed tumor growth inhibitory activity against a subset of the PPTP solid tumor and ALL xenografts. Copyright © 2011 Wiley Periodicals, Inc. DOI: 10.1002/pbc.23357 PMCID: PMC3823062 PMID: 22012946 [Indexed for MEDLINE] Conflict of interest statement: CONFLICT OF INTEREST STATEMENT: Peter G Smith, and Jie Yu are employees of Millennium Pharmaceuticals, the other authors consider that there are no actual or perceived conflicts of interest.
http://www.ncbi.nlm.nih.gov/pubmed/19355997
1. Curr Vasc Pharmacol. 2009 Apr;7(2):146-52. doi: 10.2174/157016109787455707. Infection, its treatment and the risk for stroke. Palm F(1), Urbanek C, Grau A. Author information: (1)Department of Neurology, Städtisches Klinikum Ludwigshafen, Germany. Stroke is among the most common causes of death and persisting disability and therefore represents a great social and economic burden worldwide. In order to lower this burden it is essential to identify risk factors and respective preventive strategies. Besides the established stroke risk factors (e.g. hypertension, diabetes, hypercholesterolemia, atrial fibrillation) both acute and chronic infectious diseases have emerged as risk factors for stroke. Mainly acute respiratory tract infection but also urinary tract infections independently increase the risk of ischemic stroke. Such additional risk was shown to be highest for infection within 3 days before ischemia and the risk steadily declines with increasing time intervals between infection and stroke. Associations between stroke incidence and mortality and influenza epidemics have been demonstrated. Observational studies showed an inverse association between influenza vaccination and stroke risk; however, interventional studies in this field have not been performed so far. Chronic infections, presently discussed as stroke risk factors mainly include periodontitis and infections with Helicobacter pylori (Hp) and Chlamydia pneumoniae (Cp). Although most respective studies identified these infectious diseases as independent stroke risk factors interventional trials have not been performed so far and causality is not proven, yet. There is preliminary evidence that the number of pathogens to which a subject had been exposed to rather than single pathogens are associated with the risk of stroke or other cardiovascular diseases. Chronic infectious diseases are treatable conditions and their identification as causal contributors to stroke risk could offer new avenues in stroke prevention. DOI: 10.2174/157016109787455707 PMID: 19355997 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/12147540
1. Circulation. 2002 Jul 30;106(5):580-4. doi: 10.1161/01.cir.0000023894.10871.2f. Cytotoxin-associated gene-A--positive Helicobacter pylori strains are associated with atherosclerotic stroke. Pietroiusti A(1), Diomedi M, Silvestrini M, Cupini LM, Luzzi I, Gomez-Miguel MJ, Bergamaschi A, Magrini A, Carrabs T, Vellini M, Galante A. Author information: (1)Medical Semiology and Methodology, Dipartimento di Medicina Interna, Tor Vergata University, Rome, Italy. pietroiusti@med.uniroma2.it BACKGROUND: It is uncertain whether Helicobacter pylori is associated with ischemic syndromes and whether this association is mediated by the induction of atherosclerosis. In this study, we tested the hypothesis that atherosclerotic stroke shows a selective association with virulent H pylori strains. METHODS AND RESULTS: The seroprevalence of infection by H pylori and by strains bearing the cytotoxin-associated gene-A (CagA), a strong virulence factor, was assessed by ELISA in 138 patients with large-vessel stroke (group A), in 61 patients with cardioembolic stroke (group B), and in 151 healthy control subjects. The 3 groups had a similar socioeconomic status. Serum levels of C-reactive protein were also measured by ELISA. The prevalence of infection was 71% in group A, 63.9% in group B, and 70.2% in the control group (P=NS), whereas the prevalence of CagA-positive strains was higher in group A than in group B (42.8% versus 19.7%, respectively; odds ratio 3.04, 95% CI 1.43 to 6.49; P<0.001) and higher in group A than in the control group (42.8% versus 17.9%, respectively; odds ratio 4.3, 95% CI 2.12 to 8.64; P<0.001), after adjusting for main cardiovascular risk factors and social class. A trend toward a difference in C-reactive protein was observed between CagA-positive (2.00+/-3.43 [mean+/-SD] mg/dL) and CagA-negative (1.31+/-1.72 [mean+/-SD] mg/dL) patients (P=0.072, Mann-Whitney U test). CONCLUSIONS: The association between H pylori and acute cerebrovascular disease seems to be due to a higher prevalence of more virulent H pylori strains in patients with atherosclerotic stroke. DOI: 10.1161/01.cir.0000023894.10871.2f PMID: 12147540 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/24259600
1. Ann Pharmacother. 2014 Jan;48(1):107-15. doi: 10.1177/1060028013504087. Epub 2013 Nov 1. Bedaquiline: a novel diarylquinoline for multidrug-resistant tuberculosis. Chahine EB(1), Karaoui LR, Mansour H. Author information: (1)Palm Beach Atlantic University, West Palm Beach, FL, USA. Comment in Ann Pharmacother. 2014 May;48(5):666. doi: 10.1177/1060028014521589. Ann Pharmacother. 2014 May;48(5):667. doi: 10.1177/1060028014521590. OBJECTIVE: To review the chemistry, pharmacology, microbiology, pharmacokinetics, pharmacodynamics, clinical efficacy, safety, dosage, and administration of bedaquiline, a novel oral diarylquinoline antimycobacterial agent approved by the Food and Drug Administration for the treatment of adults with pulmonary multidrug-resistant tuberculosis (MDR-TB). DATA SOURCES: A search of PubMed (January 2004-May 2013) and International Pharmaceutical Abstracts (January 2004-May 2013) using the search terms bedaquiline, diarylquinoline, R207910, and TMC207 was performed. Supplementary sources included proceedings of the Union World Conference on Lung Health. STUDY SELECTION AND DATA EXTRACTION: Preclinical data as well as Phase 1 and 2 studies published in English were evaluated. DATA SYNTHESIS: Bedaquiline possesses a unique mechanism of action that disrupts the activity of the mycobacterial adenosine triphosphate synthase. Clinical trials have been conducted evaluating the use of bedaquiline in combination with a background regimen for the treatment of adults with pulmonary MDR-TB. Bedaquiline has an excellent in vitro activity against Mycobacterium tuberculosis, including multidrug resistant M tuberculosis; however, its side effect profile limits its use against MDR-TB when no other effective regimen can be provided. Bedaquiline carries Black Box warnings for increased risk of unexplained mortality and QT prolongation. Bedaquiline is metabolized via the CYP3A4 isoenzyme and thus interacts with rifamycins and several antiretrovirals. CONCLUSIONS: In an era of emerging resistance and given the suboptimal efficacy and toxicity of currently available regimens for MDR-TB, bedaquiline represents a great addition to the existing armamentarium of anti-TB agents particularly in areas of the world where the disease is endemic. DOI: 10.1177/1060028013504087 PMID: 24259600 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/12388919
1. Med Sci Monit. 2002 Oct;8(10):CR675-84. Influence of chronic Helicobacter pylori infection on ischemic cerebral stroke risk factors. Majka J(1), Róg T, Konturek PC, Konturek SJ, Bielański W, Kowalsky M, Szczudlik A. Author information: (1)Jagiellonian University, College of Medicine, Cracow, Poland. Comment in Med Sci Monit. 2002 Dec;8(12):LE52-3; author reply LE53. BACKGROUND: Infection by Helicobacter pylori (Hp) has been linked to extradigestive pathologies including ischemic cerebral disease. The aim of our study was to assess the relationship between chronic Hp infection and ischemic stroke risk factors. MATERIAL/METHODS: 80 patients (pts) aged 60-75 years with ischemic stroke confirmed by CT scans (group I) and 80 age- and gender-matched healthy controls (group II) were included into trial. Atherosclerotic plaques from 20 Hp positive pts were obtained at carotid endarterectomy for Hp DNA assessment by PCR. In all groups following parameters were determined; 1) the prevalence of Hp infection using (13)C-Urea Breath Test (UBT), 2) plasma anti-Hp and anti-CagA IgG and interleukin-8 (IL-8), and 3) plasma lipids and fibrinogen. Hp positive pts and controls received one-week anti-Hp therapy and after six months total cholesterol, low-density lipoprotein (LDL)-cholesterol, fibrinogen and IL-8 levels were re-examined. RESULTS: Hp infection was detected by UBT in 83.75% of stroke pts but only in 65% of controls. CagA seropositivity was also significantly higher in stroke pts (57.5%) than in controls (33.75%). Plasma levels of cholesterol, LDL-cholesterol and fibrinogen as well as IL-8 were significantly higher in Hp positive subjects, especially in pts with ischemic stroke. Six months following successful anti-Hp therapy, the plasma levels of total cholesterol, LDL-cholesterol, fibrinogen and IL-8 were significantly lower than those in Hp positive stroke pts and controls. CONCLUSIONS: Hp infection represents risk factor of ischemic stoke via an interaction of Hp cytotoxins or cytokines with atherosclerotic plaques in carotic arteries. PMID: 12388919 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/18665936
1. Helicobacter. 2008 Aug;13(4):274-7. doi: 10.1111/j.1523-5378.2008.00610.x. High prevalence of Cag-A positive H. pylori strains in ischemic stroke: a primary care multicenter study. De Bastiani R(1), Gabrielli M, Ubaldi E, Benedetto E, Sanna G, Cottone C, Candelli M, Zocco MA, Saulnier N, Santoliquido A, Papaleo P, Gasbarrini G, Gasbarrini A. Author information: (1)GIGA-CP (Italian Group for Primary Care Gastroenterology), Feltre (BL) Italy. Erratum in Helicobacter. 2013 Dec;18(6):473. Zocco Maria, Assunta [corrected to Zocco, Maria Assunta]. BACKGROUND: Previous studies suggested an association between CagA-positive H. pylori strains and ischemic stroke. The aim of the present study was to assess the prevalence of Helicobacter pylori infection and CagA status in patients with atherosclerotic stroke in the primary care setting. MATERIALS AND METHODS: A total of 106 consecutive patients (age 76.6 +/- 8 years; males 52%) with well-documented history of atherosclerotic stroke and 106 sex-age- (age 76.5 +/- 9 years; males 52%) and social background-matched controls without relevant vascular diseases. Risk factors for ischemic stroke were recorded in all subjects. H. pylori infection was assessed by[13]C-urea breath test. A serologic assay for specific IgG against CagA was performed in infected subjects. RESULTS: A trend toward a higher prevalence of H. pylori was observed in cases (63%) with respect to controls (54%) without reaching a statistical significance. CagA positivity was associated to a higher risk of atherosclerotic stroke (adjusted odds ratio 2.69, 95% confidence interval 1.37-5.30). CONCLUSIONS: Our findings suggest that CagA-positive strains of H. pylori are significantly associated to atherosclerotic stroke. This is not a merely confirmative study since it has been performed for the first time in the primary care setting and included only subjects with an active infection. DOI: 10.1111/j.1523-5378.2008.00610.x PMID: 18665936 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/16709600
1. Mol Endocrinol. 2006 Oct;20(10):2444-55. doi: 10.1210/me.2006-0118. Epub 2006 May 18. Differential expression of FOXO1 and FOXO3a confers resistance to oxidative cell death upon endometrial decidualization. Kajihara T(1), Jones M, Fusi L, Takano M, Feroze-Zaidi F, Pirianov G, Mehmet H, Ishihara O, Higham JM, Lam EW, Brosens JJ. Author information: (1)Institute of Reproductive and Developmental Biology, Imperial College London, Hammersmith Hospital, London W12 0NN, United Kingdom. The integrity of the feto-maternal interface is critical for survival of the conceptus. This interface, consisting of the maternal decidua and the invading placental trophoblast, is exposed to profound changes in oxygen tension during pregnancy. We demonstrate that human endometrial stromal cells become extraordinarily resistant to oxidative stress-induced apoptosis upon decidualization in response to cAMP and progesterone signaling. This differentiation process is associated with the induction of the forkhead transcription factor FOXO1, which in turn increases the expression of the mitochondrial antioxidant manganese superoxide dismutase. However, silencing of FOXO1 did not increase the susceptibility of decidualized cells to oxidative cell death. Comparative analysis demonstrated that hydrogen peroxide, a source of free radicals, strongly induces FOXO3a mRNA and protein expression in undifferentiated human endometrial stromal cells but not in decidualized cells. Expression of a constitutively active FOXO3a mutant elicited apoptosis in decidualized cells. Furthermore, silencing of endogenous FOXO3a in undifferentiated cells abrogated apoptosis induced by hydrogen peroxide. These results suggest that the induction of FOXO1 may enhance the ability of decidualized cells to prevent oxidative damage while the simultaneous repression of FOXO3a expression disables the signaling pathway responsible for oxidative cell death. The differential regulation of FOXO expression provides the decidua with a robust system capable of coping with prolonged episodes of oxidative stress during pregnancy. DOI: 10.1210/me.2006-0118 PMID: 16709600 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/20184513
1. Amyotroph Lateral Scler. 2010;11(1-2):4-15. doi: 10.3109/17482960802379004. Dysarthria in amyotrophic lateral sclerosis: A review. Tomik B(1), Guiloff RJ. Author information: (1)Department of Neurology, Jagiellonian University, Krakow, Poland. tomik@neuro.cm-uj.krakow.pl <tomik@neuro.cm-uj.krakow.pl> Dysarthria is a motor disorder of speech characterized by abnormalities of the articulation and intelligibility of speech. Phonation and the rate of facial movements may also be affected. Understanding the nature and course of dysarthria in amyotrophic lateral sclerosis (ALS) is important because loss of communication prevents patients from participating in many activities, may lead to social isolation, and reduces the quality of life. The goal of management of dysarthria in ALS patients is to optimize communication effectiveness for as long as possible. The information about dysarthria in ALS is dispersed in physiological, pathological, speech therapy, otorhinolaringological and neurological publications. This review summarizes the current state of knowledge on the clinical features, differential diagnosis, pathophysiology, investigations and management of dysarthria in ALS patients. There is a need to compare the different methods used to assess dysarthria and for controlled clinical trials to assess therapeutic strategies. DOI: 10.3109/17482960802379004 PMID: 20184513 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23033442
1. J Speech Lang Hear Res. 2012 Oct;55(5):1472-84. doi: 10.1044/1092-4388(2012/11-0263). Characterizing intonation deficit in motor speech disorders: an autosegmental-metrical analysis of spontaneous speech in hypokinetic dysarthria, ataxic dysarthria, and foreign accent syndrome. Lowit A(1), Kuschmann A. Author information: (1)University of Strathclyde, Glasgow, Scotland. a.lowit@strath.ac.uk PURPOSE: The autosegmental-metrical (AM) framework represents an established methodology for intonational analysis in unimpaired speaker populations but has found little application in describing intonation in motor speech disorders (MSDs). This study compared the intonation patterns of unimpaired participants (CON) and those with Parkinson's disease (PD), ataxic dysarthria (AT), and foreign accent syndrome (FAS) to evaluate the approach's potential for distinguishing types of MSDs from each other and from unimpaired speech. METHOD: Spontaneous speech from 8 PD, 8 AT, 4 FAS, and 10 CON speakers were analyzed in relation to inventory and prevalence of pitch patterns, accentuation, and phrasing. Acoustic-phonetic baseline measures (maximum-phonation-duration, speech rate, and F0-variability) were also performed. RESULTS: The analyses yielded differences between MSD and CON groups and between the clinical groups in regard to prevalence, accentuation, and phrasing. AT and FAS speakers used more rising and high pitch accents than PD and CON speakers. The AT group used the highest number of pitch accents per phrase, and all 3 MSD groups produced significantly shorter phrases than the CON group. CONCLUSIONS: The study succeeded in differentiating MSDs on the basis of intonational performances by using the AM approach, thus, demonstrating its potential for charting intonational profiles in clinical populations. DOI: 10.1044/1092-4388(2012/11-0263) PMID: 23033442 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23312647
1. Handb Clin Neurol. 2013;110:273-81. doi: 10.1016/B978-0-444-52901-5.00022-8. Disorders of communication: dysarthria. Enderby P(1). Author information: (1)Department of Rehabilitation and Assistive Technology, University of Sheffield, Sheffield, UK. p.m.enderby@sheffield.ac.uk Dysarthria is a motor speech disorder which can be classified according to the underlying neuropathology and is associated with disturbances of respiration, laryngeal function, airflow direction, and articulation resulting in difficulties of speech quality and intelligibility. There are six major types of dysarthria: flaccid dysarthria associated with lower motor neuron impairment, spastic dysarthria associated with damaged upper motor neurons linked to the motor areas of the cerebral cortex, ataxic dysarthria primarily caused by cerebellar dysfunction, and hyperkinetic dysarthria and hypokinetic dysarthria, which are related to a disorder of the extrapyramidal system. The sixth is generally termed a mixed dysarthria and is associated with damage in more than one area, resulting in speech characteristics of at least two groups. The features of the speech disturbance of these six major types of dysarthria are distinctive and can assist with diagnosis. Dysarthria is a frequent symptom of many neurological conditions and is commonly associated with progressive neurological disease. It has a profound effect upon the patient and their families as communication is integrally related with expressing personality and social relationships. Speech and language therapy can be used to encourage the person to use the speech that is already available to them more effectively, can increase the range and consistency of sound production, can teach strategies for improving intelligibility and communicative effectiveness, can guide the individual to use methods that are less tiring and more successful, and can introduce the appropriate Augmentative and Alternative Communication approaches as and when required. Copyright © 2013 Elsevier B.V. All rights reserved. DOI: 10.1016/B978-0-444-52901-5.00022-8 PMID: 23312647 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/9197089
1. Folia Phoniatr Logop. 1997;49(2):63-82. doi: 10.1159/000266440. A speaking task analysis of the dysarthria in cerebellar disease. Kent RD(1), Kent JF, Rosenbek JC, Vorperian HK, Weismer G. Author information: (1)Waisman Center, University of Wisconsin-Medison 53705-2280, USA. Cerebellar disease affects a number of skilled movements, including those in speech. Ataxic dysarthria, the speech disorder that typically accompanies cerebellar disease, was studied by acoustic methods. Control subjects and subjects with ataxic dysarthria were recorded while performing a number of speaking tasks, including sustained vowel phonation, syllable repetition, monosyllabic word production (intelligibility test), sentence recitation, and conversation. Acoustic data derived from the speech samples confirmed the hypothesis that temporal dysregulation is a primary component of the speech disorder. The data also show that the nature of the disorder varies with the speaking task. This result agrees with observations on other motor systems in subjects with cerebellar disease and may be evidence of a dissociation of impairments. Suggestions are offered on the selection of measures for a given task and on the role of the cerebellum in the regulation of speaking. DOI: 10.1159/000266440 PMID: 9197089 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/20882349
1. J Inherit Metab Dis. 2011 Apr;34(2):377-85. doi: 10.1007/s10545-010-9213-4. Epub 2010 Sep 30. Voice disorders in children with classic galactosemia. Potter NL(1). Author information: (1)Department of Speech and Hearing Sciences, Washington State University-Spokane, Spokane, WA 99210-1495, USA. nlpotter@wsu.edu Children with classic galactosemia are at risk for motor speech disorders resulting from disruptions in motor planning and programming (childhood apraxia of speech or CAS) or motor execution (dysarthria). In the present study of 33 children with classic galactosemia, 21% were diagnosed with CAS, 3% with ataxic dysarthria, and 3% with mixed CAS-dysarthria. Voice disorders due to laryngeal insufficiency were common in children with dysarthria and co-occurred with CAS. Most (58%) of the children with classic galactosemia had decreased respiratory-phonatory support for speech, and 33% had disturbed vocal quality that was indicative of cerebellar dysfunction. Three children, two diagnosed with CAS and one not diagnosed with a motor speech disorder, had vocal tremors. Treatment of voice dysfunction in neurogenic speech disorders is discussed. DOI: 10.1007/s10545-010-9213-4 PMCID: PMC3063853 PMID: 20882349 [Indexed for MEDLINE] Conflict of interest statement: Competing interest: None declared
http://www.ncbi.nlm.nih.gov/pubmed/18249521
1. Presse Med. 2008 Mar;37(3 Pt 2):525-34. doi: 10.1016/j.lpm.2007.07.029. Epub 2008 Feb 4. [Does Helicobacter pylori infection play a role in extragastric diseases?]. [Article in French] de Korwin JD(1). Author information: (1)Service de médecine interne H, Université Henri Poincaré, CHU de Nancy-Hôpital Central, F-54035 Nancy Cedex, France. jd.dekorwin@chu-nancy.fr Since the discovery of Helicobacter pylori (H. pylori), numerous studies have considered the possibility that it plays a role in different extragastric diseases. Most of these studies may be classified as epidemiological studies or investigations of H. pylori eradication, but there are also case reports and in vitro studies. This review reveals the limitations common to most of them. Idiopathic thrombocytopenic purpura is the disease for which the strongest association with H. pylori infection has been shown. Data are also accumulating about the role of H. pylori infection in idiopathic iron deficiency anemia and chronic idiopathic urticaria. Interesting results show that H. pylori infection affects atherosclerosis and is weakly associated with ischemic heart disease and stroke. Moreover, CagA-positive H. pylori strains may play a role in the natural history of atherosclerotic stroke. Recent studies suggest a link between H. pylori and Parkinson's disease. Preliminary data indicate that H. pylori infection impairs gastric ghrelin production and may influence nutritional status. The association between H. pylori infection and other extragastric diseases remains controversial. H. pylori infection may cause extragastric manifestations directly or indirectly, by various mechanisms including atrophic gastritis, the release of inflammatory mediators, molecular mimicry, and systemic immune response. Evidence suggests that anti-H. pylori therapy improves idiopathic thrombocytopenic purpura (significant increase of platelet count in half of the cases), iron-deficiency anemia, and chronic urticaria (30% remission rate), but the data from randomized controlled trials are insufficient to confirm these positive effects. DOI: 10.1016/j.lpm.2007.07.029 PMID: 18249521 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/9436737
1. J Neurol Neurosurg Psychiatry. 1998 Jan;64(1):104-7. doi: 10.1136/jnnp.64.1.104. Helicobacter pylori infection: a risk factor for ischaemic cerebrovascular disease and carotid atheroma. Markus HS(1), Mendall MA. Author information: (1)Department of Clinical Neuroscience, King's College School of Medicine and Dentistry and the Institute of Psychiatry, London, UK. h.markus@iop.bpmf.ac.uk OBJECTIVES: Chronic Helicobacter pylori infection has been associated with ischaemic heart disease although the mechanism by which it mediates this effect remains unclear. The objective was to determine whether it is also a risk factor for ischaemic cerebrovascular disease METHODS: A total of 238 patients and 119 controls were studied. Patients were characterised into stroke subtypes based on pathogenic mechanisms and carotid atheroma load was estimated using duplex ultrasound. H pylori seropositivity was determined on serum samples. RESULTS: H pylori seropositivity was more common in cases (58.8% v 44.5%, p=0.01). The odds ratio for cerebrovascular disease associated with seropositivity was 1.78 (95% confidence interval (95% CI) 1.14-2.77), and this remained significant after controlling for other risk factors including socioeconomic status (1.63 (95% CI 1.02-2.60). H pylori seropositivity was associated with large vessel disease (odds ratio 2.58 (95% CI 1.44-4.63), p=0.001) and lacunar stroke (odds ratio 2.21 (95% CI 1.12-4.38), p=0.02) but not stroke due to cardioembolism or unknown aetiology (odds ratio 1.16 (95% CI 0.66-2.02), p=0.5). Mean (SD) carotid stenosis was greater in patients seropositive for H pylori (37.3 (29.7) v 27.9 (26.2)%, p=0.01). There was no difference in the prevalence of seropositivity between patients with stroke and transient ischaemic attack (59.6% v 58.6%, p=0.9) CONCLUSION: Chronic H pylori infection is an independent risk factor for ischaemic cerebrovascular disease and may act, at least in part, by increasing atherosclerosis. DOI: 10.1136/jnnp.64.1.104 PMCID: PMC2169914 PMID: 9436737 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/12725602
1. Dig Liver Dis. 2003 Jan;35(1):16-9. doi: 10.1016/s1590-8658(02)00005-1. Helicobacter pylori infection in subjects with acute ischaemic stroke. Moayyedi P(1), Carter AM, Braunholtz D, Catto AJ. Author information: (1)Unit of Molecular Vascular Medicine, Research School of Medicine, University of Leeds, General Infirmary at Leeds, Leeds LS1 3EX, UK. p.moayyedi@bham.ac.uk AIMS: To determine whether infection with Helicobacter pylori is a significant risk factor for stroke. SUBJECTS: A total 467 in-patients with clinical evidence of acute ischaemic stroke and 388 healthy controls with no evidence of cerebrovascular disease. METHODS: This was a case control study. The prevalence of Helicobacter pylori was measured by enzyme-linked immunosorbent assay in stroke patients and controls. A positive titre was defined as >15 U/ml and relationship with circulating plasma fibrinogen and social depravation was expressed using the Townsend Index. RESULTS: There were significantly more Helicobacter pylori positive individuals (274/398 (69%)) in the cases compared to the controls (206/352 (58.5%)). Fibrinogen levels were also significantly higher in Helicobacter pylori positive (mean 4.14, standard deviation 1.33) than negative individuals (mean 3.78, standard deviation 1.28). The association between Helicobacter pylori and stroke was lost in a logistic model controlling for socio-economic status. Furthermore, fibrinogen levels were not associated with Helicobacter pylori status in a linear regression model controlling for socio-economic status. CONCLUSIONS: Infection with Helicobacter pylori is associated with an increased risk of stroke and increased fibrinogen levels but these findings can be attributed to a confounding effect of socio-economic status. DOI: 10.1016/s1590-8658(02)00005-1 PMID: 12725602 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/14500942
1. Stroke. 2003 Oct;34(10):2518-32. doi: 10.1161/01.STR.0000089015.51603.CC. Epub 2003 Sep 18. Inflammation and infections as risk factors for ischemic stroke. Lindsberg PJ(1), Grau AJ. Author information: (1)Department of Neurology, Helsinki University Central Hospital and Biomedicum Helsinki, Helsinki, Finland. perttu.lindsberg@hus.fi BACKGROUND: Inflammatory processes have fundamental roles in stroke in both the etiology of ischemic cerebrovascular disease and the pathophysiology of cerebral ischemia. We summarize clinical data on infection and inflammation as risk or trigger factors for human stroke and investigate current evidence for the hypothesis of a functional interrelation between traditional risk factors, genetic predisposition, and infection/inflammation in stroke pathogenesis. SUMMARY OF REVIEW: Several traditional vascular risk factors are associated with proinflammatory alterations, including leukocyte activation, and predispose cerebral vasculature to thrombogenesis on inflammatory stimulation. Furthermore, accumulation of inflammatory cells, mainly monocytes/macrophages, within the vascular wall starts early during atherogenesis. During later disease stages, their activation can lead to plaque rupture and thrombus formation, increasing stroke risk. Inflammatory markers (eg, leukocytes, fibrinogen, C-reactive protein) are independent predictors of ischemic stroke. Chronic infections (eg, infection with Chlamydia pneumoniae or Helicobacter pylori) were found to increase the risk of stroke; however, study results are at variance, residual confounding is not excluded, and causality is not established at present. In case-control studies, acute infection within the preceding week was a trigger factor for ischemic stroke. Acute and exacerbating chronic infection may act by activating coagulation and chronic infections and may contribute to atherogenesis. Genetic predisposition of the inflammatory host response may be an important codeterminant for atherogenesis and stroke risk. CONCLUSIONS: Inflammation contributes to stroke risk via various interrelated mechanisms. Infectious diseases, traditional risk factors, and genetic susceptibility may cooperate in stimulating inflammatory pathways. Final proof of a causal role of infectious/inflammatory mechanisms in stroke pathogenesis is still lacking and will require interventional studies. DOI: 10.1161/01.STR.0000089015.51603.CC PMID: 14500942 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/11412864
1. J Neurol Sci. 2001 May 1;186(1-2):1-5. doi: 10.1016/s0022-510x(01)00507-x. Helicobacter pylori infection as an independent risk factor for cerebral ischemia of atherothrombotic origin. Grau AJ(1), Buggle F, Lichy C, Brandt T, Becher H, Rudi J. Author information: (1)Department of Neurology, University of Heidelberg, Im Neuenheimer Feld 400, 69120 Heidelberg, Germany. Armin_Grau@med.uni-heidelberg.de Chronic infection may increase the risk for ischemic stroke. Presently, it is insufficiently established whether Helicobacter pylori infection represents a risk factor for ischemic stroke. We analyzed IgG antibodies against H. pylori in 109 patients with acute cerebral ischemia and 82 age- and sex-matched control patients with non-vascular and non-inflammatory neurological diseases. Antibody titers were significantly higher in patients than in control subjects (p=0.007). H. pylori seropositivity tended to be more common in patients (odds ratio (OR) 1.55, 95% confidence interval (ci) 0.87-2.76), but this trend was further attenuated in multivariate analysis (OR 1.42; 95% 0.75-2.67) with hypertension, diabetes mellitus, current or previous smoking, previous cerebral ischemia and low socioeconomic status. H. pylori seropositivity increased the odds for cerebral ischemia of atherothrombotic origin in univariate (OR 3.63; 95% ci 1.37-9.65) and multivariate analysis (OR 3.53; 95% ci 1.09-11.4). H. pylori seropositivity may be an independent risk factor for stroke of atherothrombotic origin. DOI: 10.1016/s0022-510x(01)00507-x PMID: 11412864 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/16466297
1. Expert Rev Neurother. 2006 Feb;6(2):175-83. doi: 10.1586/14737175.6.2.175. The effect of infections and vaccinations on stroke risk. Grau AJ(1), Marquardt L, Lichy C. Author information: (1)Department of Neurology, Klinikum der Stadt Ludwigshafen, Bremserstr. 79, 67063 Ludwigshafen a. Rh., Germany. graua@klilu.de There is increasing evidence that, in addition to conventional risk factors, acute and chronic infectious diseases increase the risk of stroke. Acute infection, mainly respiratory, and both bacterial and viral infection, represent temporarily active trigger factors for cerebral ischemia. Chronic infectious diseases that may increase the risk of stroke include periodontitis, chronic bronchitis and infections with microbial antigens, such as Helicobacter pylori and Chlamydia pneumoniae. From observational studies, there is evidence that vaccination against influenza is associated with a reduced risk of stroke, myocardial infarction and all-cause mortality. This report provides an overview on the influence of infection on stroke risk and potential anti-infective strategies that may play a future role in stroke prevention. DOI: 10.1586/14737175.6.2.175 PMID: 16466297 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23237251
1. Expert Rev Gastroenterol Hepatol. 2012 Dec;6(6):667-74. doi: 10.1586/egh.12.47. LINX(™) Reflux Management System: magnetic sphincter augmentation in the treatment of gastroesophageal reflux disease. Bonavina L(1), DeMeester TR, Ganz RA. Author information: (1)Department of Biomedical Sciences for Health, University of Milano Medical School, Division of General Surgery, IRCCS Policlinico San Donato, Via Morandi 30 20097, San Donato Milanese, Milan, Italy. luigi.bonavina@unimi.it Gastroesophageal reflux disease (GERD), commonly manifested by heartburn or regurgitation, is a chronic, progressive condition in which failed sphincter function allows the contents of the stomach to reflux into the esophagus, the airways and the mouth. Chronic GERD affects 10% of Western society. The majority of patients receive adequate relief from proton pump inhibitors, but up to 40% have incomplete relief of symptoms that cannot be addressed by increasing the dose of medications. The laparoscopic Nissen fundoplication is the surgical gold standard; however, the level of technical difficulty and its side effects have limited its use to less than 1% of the GERD population. These factors have contributed to the propensity of patients to persist with medical therapy, even when inadequate to control symptoms and complications of the disease. Consequently, a significant gap in the treatment continuum for GERD remains evident in current clinical practice. The LINX(™) Reflux Management System (Torax Medical) is designed to provide a permanent solution to GERD by augmenting the physiologic function of the sphincter barrier with a simple and reproducible laparoscopic procedure that does not alter gastric anatomy and can be easily reversed if necessary. DOI: 10.1586/egh.12.47 PMID: 23237251 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/18706211
1. Chin Med J (Engl). 2008 May 20;121(10):946-51. Cytotoxin-associated gene-A-seropositive virulent strains of Helicobacter pylori and atherosclerotic diseases: a systematic review. Zhang S(1), Guo Y, Ma Y, Teng Y. Author information: (1)Department of Neurology, Shengjing Hospital, China Medical University, Shenyang, Liaoning 110004, China. OBJECTIVE: A systematic meta-analysis was performed to explore the role of cytotoxin-associated gene-A (CagA) seropositive strains of Helicobacter pylori (H. pylori) in the pathogenesis of atherosclerotic diseases. Data sources Data from Medline, EMBASE, CBMdisc, CNKI and the Cochrane Collaboration database were searched. Similar search strategies were applied to each of these databases. Study selection The review was restricted to the case-control studies on infective, chronic virulent CagA strains of H. pylori, involving the risk of ischemic stroke and coronary heart disease, ineligible studies were excluded. Two reviewers independently extracted the data and assessed study quality. RESULTS: Totally 26 case-control studies (11 studies on ischemic stroke and 15 studies on coronary heart disease) were retrieved and considered. The combined data revealed that the chronic seropositive virulent strains of H. pylori infection had a trend of increasing the risk of ischemic strokes and coronary heart diseases, yielding pooled ORs of 2.68 (95% CI: 2.20, 3.27) and 2.11 (95% CI: 1.70, 2.62), respectively. We also performed subgroup analyses, dividing the total population into Caucasian and Chinese subgroups. Through the subgroup analysis, no significant difference was found between the subgroups. CONCLUSIONS: Our results support the hypothesis that CagA-seropositive strains infection is significantly associated with susceptibility to ischemic strokes and coronary heart diseases. The magnitude of the association with atherosclerotic diseases needs to be confirmed by prospective studies and the studies on CagA-seropositive strains eradication are more important. PMID: 18706211 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/8068159
1. Pediatr Neurol. 1994 Jun;10(4):317-9. doi: 10.1016/0887-8994(94)90129-5. Normal serum beta-galactosidase in juvenile GM1 gangliosidosis. Ishii N(1), Oshima A, Sakuraba H, Fukuyama Y, Suzuki Y. Author information: (1)Department of Clinical Genetics, Tokyo Metropolitan Institute of Medical Science, Japan. GM1 gangliosidosis is a genetic disease with lysosomal beta-galactosidase deficiency caused by mutations of the gene coding for this enzyme. However, apparently normal enzyme activity was found in plasma or serum from juvenile GM1 gangliosidosis patients homozygous for a mutation, R201C (201Arg-->Cys), after clotting for 30 min. This extracellular fluid finding is unusual in patients with primary and genetic deficiency of beta-galactosidase. The serum enzyme activity became relatively low only after 3 1/2-hour clotting because its increase in normal controls was not observed in these patients. beta-Galactosidase assay is not always reliable, particularly with serum or plasma as an enzyme source, for the diagnosis of hereditary beta-galactosidase deficiency, unless it is conducted under well-controlled conditions. DOI: 10.1016/0887-8994(94)90129-5 PMID: 8068159 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/15166387
1. Stroke. 2004 Aug;35(8):1800-4. doi: 10.1161/01.STR.0000131751.35926.48. Epub 2004 May 27. Association between cerebral ischemia and cytotoxin-associated gene-A-bearing strains of Helicobacter pylori. Preusch MR(1), Grau AJ, Buggle F, Lichy C, Bartel J, Black C, Rudi J. Author information: (1)Department of Neurology, University of Heidelberg, Germany. BACKGROUND AND PURPOSE: Studies on Helicobacter pylori infection and risk of ischemic stroke yielded variable results. Infection with more virulent H. pylori strains, such as cytotoxin-associated gene-A (CagA)-bearing strains, may be of particular relevance for ischemic diseases. We investigated whether H. pylori and CagA seropositivity are independent risk factors for cerebral ischemia or its etiologic subtypes. METHODS: We determined IgG antibodies against H. pylori and CagA protein (enzyme immunoassays) in 190 patients with acute cerebral ischemia and in 229 age- and sex-matched control subjects selected randomly from the general population. RESULTS: CagA seropositivity was more common in patients (114/190; 60.0%) than in control subjects (99/229; 43.2%; odds ratio, 1.97; 95% CI, 1.33 to 2.91; P<0.001). This result remained significant after adjustment for age, sex, vascular risk factors and diseases, and childhood and adult social status (odds ratio, 1.84; 95% CI, 1.13 to 3.00; P=0.015). Subgroup analyses yielded similar results in all etiologic stroke subtypes. In contrast, H. pylori seropositivity in general was not associated with increased risk of stroke or its etiologic subtypes. CONCLUSIONS: Our results support the hypothesis of an association between infection with CagA-positive H. pylori strains and acute cerebral ischemia. DOI: 10.1161/01.STR.0000131751.35926.48 PMID: 15166387 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/10571006
1. Biochim Biophys Acta. 1999 Oct 8;1455(2-3):85-103. doi: 10.1016/s0925-4439(99)00075-7. Molecular basis of GM1 gangliosidosis and Morquio disease, type B. Structure-function studies of lysosomal beta-galactosidase and the non-lysosomal beta-galactosidase-like protein. Callahan JW(1). Author information: (1)Department of Pediatric Laboratory Medicine, The Hospital for Sick Children, University of Toronto, ON, Canada. jwc@sickkids.on.ca GM1 gangliosidosis and Morquio B disease are distinct disorders both clinically and biochemically yet they arise from the same beta-galactosidase enzyme deficiency. On the other hand, galactosialidosis and sialidosis share common clinical and biochemical features, yet they arise from two separate enzyme deficiencies, namely, protective protein/cathepsin A and neuraminidase, respectively. However distinct, in practice these disorders overlap both clinically and biochemically so that easy discrimination between them is sometimes difficult. The principle reason for this may be found in the fact that these three enzymes form a unique complex in lysosomes that is required for their stability and posttranslational processing. In this review, I focus mainly on the primary and secondary beta-galactosidase deficiency states and offer some hypotheses to account for differences between GM1 gangliosidosis and Morquio B disease. DOI: 10.1016/s0925-4439(99)00075-7 PMID: 10571006 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/19842039
1. Apoptosis. 2010 Jan;15(1):83-93. doi: 10.1007/s10495-009-0415-x. Mouse 3T3-L1 cells acquire resistance against oxidative stress as the adipocytes differentiate via the transcription factor FoxO. Kojima T(1), Norose T, Tsuchiya K, Sakamoto K. Author information: (1)Graduate School of Life and Environmental Sciences, University of Tsukuba, Tsukuba, Ibaraki 305-8572, Japan. Repression of excessive increase and enlargement of adipocytes that is closely associated with obesity is effective in the prevention and treatment of metabolic syndrome. Generally, apoptosis is induced in cells via a wide variety of intracellular or extracellular substances, and recently, it has been suggested that the FoxO subfamily is involved in the induction of apoptosis. We aimed to elucidate the mechanism of FoxO-mediated apoptosis-induction in the adipocytes under the reactive oxygen species (ROS) stimulus. The treatment of differentiated and undifferentiated 3T3-L1 cells with glucose oxidase (GOD), an enzyme that generates H(2)O(2), induced apoptosis and led to the accumulation of 8-OHdG. Apoptosis analysis revealed that GOD treatment induced apoptosis in differentiated 3T3-L1 cells less efficiently than in undifferentiated preadipocytes. GOD remarkably increased the levels of Bad, Bax, and Bim-the genes that are actively involved in cell apoptosis. GOD treatment also increased the expression of FoxO3a mRNA and protein. The introduction of FoxO3a-siRNA into 3T3-L1 cells suppressed the oxidative stress-induced expression of Bim mRNA, as well as the GOD-induced apoptosis. Furthermore, the expression of MnSOD, Cu/ZnSOD, and catalase, as well as of FoxO, increased significantly along with the progression of adipocyte differentiation. These results indicated that ROS-induced apoptosis in undifferentiated 3T3-L1 cells via the expression of FoxO3a, whereas FoxO expression suppressed the ROS-induced apoptosis in differentiated 3T3-L1 cells via the expression of ROS-scavenging enzymes. DOI: 10.1007/s10495-009-0415-x PMID: 19842039 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/1809779
1. J Commun Disord. 1991 Oct-Dec;24(5-6):393-409. doi: 10.1016/0021-9924(91)90011-7. Dysarthria of motor neuron disease: longitudinal measures of segmental durations. Seikel JA(1), Wilcox KA, Davis J. Author information: (1)Department of Speech and Hearing Sciences, Washington State University, Pullman 99164-2420. Erratum in J Commun Disord 1992 Apr-Jun;25(2-3):201-3. Motor neuron disease encompasses a group of terminal, demyelinating diseases affecting upper- and lower-motor neurons and producing muscular weakness resulting in a flaccid, spastic, or spastic-flaccid dysarthria of speech. The present study presents measurements of the temporal-acoustic characteristics of dysarthria in three subjects with Motor Neuron Disease over a two-year recording period. Changes seen over the course of the disease varied by type of motor neuron disease, though all types demonstrated some degree of neutralization of the prevocalic VOT, target vowel duration, and postvocalic closure duration. These changes are discussed with relation to physical manifestation and progression of the disease. DOI: 10.1016/0021-9924(91)90011-7 PMID: 1809779 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22774423
1. Span J Psychol. 2012 Jul;15(2):495-504. doi: 10.5209/rev_sjop.2012.v15.n2.38860. Differential diagnosis between apraxia and dysarthria based on acoustic analysis. Melle N(1), Gallego C. Author information: (1)Departamento Psicología Básica II, Facultad de Psicología, Universidad Complutense Madrid, Spain. nmelle@psi.ucm.es Acoustic analysis provides objective quantitative measures of speech that enable a comprehensive and accurate understanding of motor disorders and complement the traditional measures. This paper aims to distinguish between normal and pathological speech, more specifically between apraxia of speech and spastic dysarthria in native Spanish speaking patients using acoustic parameters. Participants (4 aphasic with apraxia of speech, 4 with spastic dysarthria, and 15 without speech disorders) performed three different tasks: repeating the syllable sequence [pa-ta-ka], repeating the isolated syllable [pa] and repeating the vowel sequence [i-u]. The results showed that the normative values of motor control, in general, coincide with those obtained in previous research on native English speakers. They also show that damage to motor control processes results in a decrease in the rate of alternating and sequential movements and an increase in the inter-syllabic time for both types of movements. A subset of the acoustic parameters analyzed, those that measure motor planning processes, enable differentiation between normal population and apraxic and dysarthric patients, and between the latter. The differences between the pathological groups support the distinction between motor planning and motor programming as described by van der Merwe's model of sensorimotor processing (1997). DOI: 10.5209/rev_sjop.2012.v15.n2.38860 PMID: 22774423 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23155537
1. Zhonghua Wei Chang Wai Ke Za Zhi. 2012 Sep;15(9):889-92. [Treatment of gastroesophageal reflux disease: comments from thoracic surgeon]. [Article in Chinese] Li ZG(1). Author information: (1)Department of ThorocicSurgery, Changhai Hospital, Shanghai, China. dr_lizhigang@hotmail.com Gastroesophageal reflux disease (GERD) is the most common gastrointestinal diagnosis recorded during visits to outpatient clinics in west countries. The prevalence of symptom-defined GERD in China is as high as 3% to 5%. Asa dysfunction, GERD is characterized by reflux and heartburn. The pathophysiologic process of GERD is very complicated and subtle. The spectrum of injury from long-term reflux of acid or bile includes damage mucosa, Barrett's esophagus, dysplasia, and esophageal cancer. Therefore, the therapies of GERD should focus on controlling symptom,treating complications, and surveillance the possibility of oncologic transform. As with therapy with proton-pump inhibitors (PPI), modifying lifestyle is another most important modality for most GERD. The window of surgical treatment for GERD is narrow. Surgical therapy is alternative management approach to the patients with PPI failure, complications, or huge hernia. The laparoscopic minimally invasive procedure improves the acceptance of patients to surgical therapy, but the long-term complication and drawbacks of anti-reflux surgery cannot be ignored, and which is even more common than open procedures. The limitations of current therapy for GERD have encouraged a search for more effective treatment.The Linx sphincter augmentation device has been developed to address this gap with improvement of the barrier function of LES and reversible design if necessary. PMID: 23155537 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/8112731
1. Hum Genet. 1994 Feb;93(2):109-14. doi: 10.1007/BF00210592. Intracellular processing and maturation of mutant gene products in hereditary beta-galactosidase deficiency (beta-galactosidosis). Oshima A(1), Yoshida K, Itoh K, Kase R, Sakuraba H, Suzuki Y. Author information: (1)Department of Clinical Genetics, Tokyo Metropolitan Institute of Medical Science, Japan. Heterogeneous patterns of biosynthesis, posttranslational processing, and degradation were demonstrated for mutant enzymes in three clinical forms of beta-galactosidase deficiency (beta-galactosidosis): juvenile GM1-gangliosidosis, adult GM1-gangliosidosis, and Morquio B disease. The precursor of the mutant enzyme in adult GM1-gangliosidosis was not phosphorylated, and only a small portion of the gene product reached the lysosomes. The enzyme in Morquio B disease was normally processed and transported to lysosomes, but its catalytic activity was low. A common gene mutation in juvenile GM1-gangliosidosis (R201C) produced an enzyme protein that did not aggregate with protective protein in the lysosome, and was rapidly degraded by thiol proteases. This abnormal turnover was similar to that for the normal but dissociated beta-galactosidase in galactosialidosis. Protease inhibitors restored the enzyme activity in fibroblasts of this clinical form. A possible therapeutic approach is discussed for this specific type of enzyme deficiency. DOI: 10.1007/BF00210592 PMID: 8112731 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/17442056
1. J Neurochem. 2007 Jun;101(5):1294-302. doi: 10.1111/j.1471-4159.2007.04525.x. Epub 2007 Apr 17. Neurochemical, morphological, and neurophysiological abnormalities in retinas of Sandhoff and GM1 gangliosidosis mice. Denny CA(1), Alroy J, Pawlyk BS, Sandberg MA, d'Azzo A, Seyfried TN. Author information: (1)Biology Department, Boston College, Chestnut Hill, Massachusetts 02467, USA. Retinal abnormalities are well documented in patients with ganglioside storage diseases. The total content and distribution of retinal glycosphingolipids was studied for the first time in control mice and in Sandhoff disease (SD) and GM1 gangliosidosis mice. Light and electron microscopy of the SD and the GM1 retinas revealed storage in ganglion cells. Similar to previous findings in rat retina, GD3 was the major ganglioside in mouse retina, while GM2 and GM1 were minor species. Total ganglioside content was 44% and 40% higher in the SD and the GM1 retinas, respectively, than in the control retinas. Furthermore, GM2 and GM1 content were 11-fold and 51-fold higher in the SD and the GM1 retinas than in the control retinas, respectively. High concentrations of asialo-GM2 and asialo-GM1 were found in the SD and the GM1 retinas, respectively, but were undetectable in the control retinas. The GSL abnormalities in the SD and the GM1 retinas reflect significant reductions in beta-hexosaminidase and beta-galactosidase enzyme activities, respectively. Although electroretinograms appeared normal in the SD and the GM1 mice, visual evoked potentials were subnormal in both mutants, indicating visual impairments. Our findings present a model system for assessing retinal pathobiology and therapies for the gangliosidoses. DOI: 10.1111/j.1471-4159.2007.04525.x PMID: 17442056 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/1909089
1. Am J Hum Genet. 1991 Sep;49(3):566-74. GM1-gangliosidosis (genetic beta-galactosidase deficiency): identification of four mutations in different clinical phenotypes among Japanese patients. Nishimoto J(1), Nanba E, Inui K, Okada S, Suzuki K. Author information: (1)Department of Neurology, University of North Carolina School of Medicine, Chapel Hill 27599-7250. GM1-gangliosidosis is a genetic neurological disorder caused by mutations in the lysosomal acid beta-galactosidase gene. While its phenotypic expression is complex, it is usually classified as being of infantile, juvenile, or adult form, on the basis of age at onset, the rate of symptomatic progression, and severity of central nervous system involvement. We have analyzed the acid beta-galactosidase gene in 12 Japanese patients from nine families. The aim was to identify mutations in individual patients and then to examine possible correlation between the mutations and the clinical phenotypes. Northern blotting studies with a full-length human beta-galactosidase cDNA showed that the mRNA ranged from undetectable to substantially decreased in the infantile patients but was normal in quantity and size in all juvenile and adult patients. Four distinct missense mutations have been identified, each limited to the respective clinical forms within our small-size samples. In the infantile patient with decreased but detectable mRNA, a point mutation was found resulting in Arg49----Cys. In the infantile patient with nearly undetectable mRNA, mutation Arg457----Ter was identified. The mutation Arg201----Cys was found in all four of the juvenile patients, while all six adult patients were homozygous for the point mutation Ile51----Thr. The mutations found in the juvenile and adult patients alter restriction sites in the normal gene and thus are amendable to quick screening. The prediction that these mutations are responsible for the clinical disease was confirmed by no expression of the catalytic activity of the mutant proteins in the COS-I cell expression system.(ABSTRACT TRUNCATED AT 250 WORDS) PMCID: PMC1683129 PMID: 1909089 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22967682
1. BMJ Case Rep. 2012 Sep 11;2012:bcr2012006669. doi: 10.1136/bcr-2012-006669. Dyke-Davidoff-Masson syndrome-like picture in a case of Takayasu arteritis: an enigma. Roy K(1), Talukdar A, Ray S, Pal P. Author information: (1)Department of General Medicine, Medical College, Kolkata, India. Authors describe the case of a 16-year-old girl who presented with fever, tonic-clonic seizures, unequal arm blood pressures and pulselessness in the left upper limb. On examination, there was a systolic bruit over umbilical region, a pansystolic murmur of mitral regurgitation was found. Neurological examination was normal except for an asymmetry in brain hemicircumference one side compared with the other. She has borderline intelligence (IQ 70) according to Wechsler Adult Performance Intelligence Scale. Magnetic resonance imaging (MRI) of brain revealed atrophic of left cerebral hemisphere with mildly ventricular dilatation, prominent paranasal and mastoid air cells, suggestive of Dyke-Davidoff-Masson syndrome (DDMS). Conventional angiography showed narrowed left internal carotid artery. There was also stenosed brachial artery, absent left renal artery with narrowed infrarenal abdominal aorta. The patient was put on antihypertensive drugs. We hypothesise that Takayasu arteritis and related vascular occlusion is the cause of her acquired cerebral changes. DOI: 10.1136/bcr-2012-006669 PMCID: PMC4543674 PMID: 22967682 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22538694
1. Surg Endosc. 2012 Oct;26(10):2944-9. doi: 10.1007/s00464-012-2289-1. Epub 2012 Apr 27. The LINX® reflux management system: confirmed safety and efficacy now at 4 years. Lipham JC(1), DeMeester TR, Ganz RA, Bonavina L, Saino G, Dunn DH, Fockens P, Bemelman W. Author information: (1)Department of Surgery, University of Southern California, 1510 San Pablo Street, HCC-514, Los Angeles, CA 90033, USA. jlipham@surgery.usc.edu BACKGROUND: Sphincter augmentation with the LINX® Reflux Management System is a surgical option for patients with chronic gastroesophageal disease (GERD) and an inadequate response to proton pump inhibitors (PPIs). Clinical experience with sphincter augmentation is now available out to 4 years. METHODS: In a multicenter, prospective, single-arm study, 44 patients underwent a laparoscopic surgical procedure for placement of the LINX System around the gastroesophageal junction (GEJ). Each patient's baseline GERD status served as the control for evaluations post implant. Long-term efficacy measures included esophageal acid exposure, GERD quality-of-life measures, and use of PPIs. Adverse events and long-term complications were closely monitored. RESULTS: For esophageal acid exposure, the mean total % time pH < 4 was reduced from 11.9 % at baseline to 3.8 % at 3 years (p < 0.001), with 80 % (18/20) of patients achieving pH normalization (≤ 5.3 %). At ≥ 4 years, 100 % (23/23) of the patients had improved quality-of-life measures for GERD, and 80 % (20/25) had complete cessation of the use of PPIs. There have been no reports of death or long-term device-related complications such as migration or erosion. CONCLUSIONS: Sphincter augmentation with the LINX Reflux Management System provided long-term clinical benefits with no safety issues, as demonstrated by reduced esophageal acid exposure, improved GERD-related quality of life, and cessation of dependence on PPIs, with minimal side effects and no safety issues. Patients with inadequate symptom control with acid suppression therapy may benefit from treatment with sphincter augmentation. DOI: 10.1007/s00464-012-2289-1 PMID: 22538694 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/10757351
1. Hum Gene Ther. 2000 Mar 20;11(5):715-27. doi: 10.1089/10430340050015617. Correction of acid beta-galactosidase deficiency in GM1 gangliosidosis human fibroblasts by retrovirus vector-mediated gene transfer: higher efficiency of release and cross-correction by the murine enzyme. Sena-Esteves M(1), Camp SM, Alroy J, Breakefield XO, Kaye EM. Author information: (1)Molecular Neurogenetics Unit, Massachusetts General Hospital, Harvard Medical School, Boston 02129, USA. Mutations in the lysosomal acid beta-galactosidase (EC 3.2.1.23) underlie two different disorders: GM1 gangliosidosis, which involves the nervous system and visceral organs to varying extents, and Morquio's syndrome type B (Morquio B disease), which is a skeletal-connective tissue disease without any CNS symptoms. This article shows that transduction of human GM1 gangliosidosis fibroblasts with retrovirus vectors encoding the human acid beta-galactosidase cDNA leads to complete correction of the enzymatic deficiency. The newly synthesized enzyme is correctly processed and targeted to the lysosomes in transduced cells. Cross-correction experiments using retrovirus-modified cells as enzyme donors showed, however, that the human enzyme is transferred at low efficiencies. Experiments using a different retrovirus vector carrying the human cDNA confirmed this observation. Transduction of human GM1 fibroblasts and mouse NIH 3T3 cells with a retrovirus vector encoding the mouse beta-galactosidase cDNA resulted in high levels of enzymatic activity. Furthermore, the mouse enzyme was found to be transferred to human cells at high efficiency. Enzyme activity measurements in medium conditioned by genetically modified cells suggest that the human beta-galactosidase enzyme is less efficiently released to the extracellular space than its mouse counterpart. This study suggests that lysosomal enzymes, contrary to the generalized perception in the field of gene therapy, may differ significantly in their properties and provides insights for design of future gene therapy interventions in acid beta-galactosidase deficiency. DOI: 10.1089/10430340050015617 PMID: 10757351 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/2837434
1. Hum Hered. 1988;38(2):76-82. doi: 10.1159/000153762. Lysosomal enzyme activities among Chinese: leukocyte alpha-galactosidase and beta-galactosidase. Xu YK(1), Ng WG. Author information: (1)Department of Pediatrics, University of Southern California School of Medicine, Los Angeles. alpha-Galactosidase and beta-galactosidase activities have been determined in leukocyte preparations from 100 randomly selected Chinese adults. For alpha-galactosidase, two groups with low activities were identified: group I consisted of 3 females having activities below 40% of normal, and group II consisted of 5 males and 1 female with activities about 60% of normal. Family studies suggested that these low alpha-galactosidase activities are genetically determined. Only 1 individual was found to have about 50% of normal beta-galactosidase activity; presumably he is a carrier for beta-galactosidase deficiency (GM1 gangliosidosis). DOI: 10.1159/000153762 PMID: 2837434 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/25264655
1. Am Surg. 2014 Oct;80(10):1034-8. Magnetic sphincter augmentation with the LINX device for gastroesophageal reflux disease after U.S. Food and Drug Administration approval. Reynolds JL(1), Zehetner J, Bildzukewicz N, Katkhouda N, Dandekar G, Lipham JC. Author information: (1)Keck Medical Center of the University of Southern California, Los Angeles, California, USA. Magnetic sphincter augmentation (MSA) of the gastroesophageal junction with the LINX Reflux Management System is an alternative to fundoplication for gastroesophageal reflux disease (GERD) that was approved by the U.S. Food and Drug Administration (FDA) in March 2012. This is a prospective observational study of all patients who underwent placement of the LINX at two institutions from April 2012 to December 2013 to evaluate our clinical experience with the LINX device after FDA approval. There were no intraoperative complications and only four mild postoperative morbidities: three urinary retentions and one readmission for dehydration. The mean operative time was 60 minutes (range, 31 to 159 minutes) and mean length of stay was 11 hours (range, 5 to 35 hours). GERD health-related quality-of-life scores were available for 83 per cent of patients with a median follow-up of five months (range, 3 to 14 months) and a median score of four (range, 0 to 26). A total of 76.9 per cent of patients were no longer taking proton pump inhibitors. The most common postoperative complaint was dysphagia, which resolved in 79.1 per cent of patients with a median time to resolution of eight weeks. There were eight patients with persistent dysphagia that required balloon dilation with improvement in symptoms. MSA with LINX is a safe and effective alternative to fundoplication for treatment of GERD. The most common postoperative complaint is mild to moderate dysphagia, which usually resolves within 12 weeks. PMID: 25264655 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/3088302
1. Jpn J Exp Med. 1986 Feb;56(1):1-11. Abnormalities of cerebral lipids in GM1-gangliosidoses, infantile, juvenile, and chronic type. Kasama T, Taketomi T. Cerebral lipids of patients with GM1-gangliosidoses, infantile, juvenile, and chronic type which are caused by deficiency of beta-galactosidase, were examined and compared to each other. The infantile type demonstrated abnormal accumulation of GM1 and asialo-GM1 in contrast with marked decrease in such major cerebral lipids as cholesterol, phospholipids, cerebroside, and sulfatide. It was also noted that significant amounts of such unusual lipids as free fatty acids, GlcCer, LacCer, GbOse3Cer, and GbOse-4Cer plus nLcOse4Cer were found in the brain. These findings pointed out that this infantile type might accompany a severe cerebral dysgenesis with poor myelination. The juvenile type also showed marked increase in GM1 and asialo-GM1, but the decrease in cholesterol, phospholipids, cerebroside, and sulfatide was not so much as the infantile type. These findings along with the occurrence of cholesterol ester suggested that the brain caused progressive demyelination after the immature myelin appeared. An autopsized brain tissue of a male patient who was eventually diagnosed as a case of GM1-gangliosidosis chronic type after his death, showed some accumulation of GM1 and asialo-GM1 particularly in the caudate nucleus and putamen, whereas it showed moderate amounts of GM1 in apparently normal gray and white matters. It seemed that there are no abnormal cerebral lipids except for gangliosides and some neutral glycosphingolipids in the chronic type. PMID: 3088302 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23622392
1. Handb Clin Neurol. 2013;113:1707-8. doi: 10.1016/B978-0-444-59565-2.00039-3. Gangliosidoses. Patterson MC(1). Author information: (1)Division of Child and Adolescent Neurology, Mayo Clinic, Rochester, MN, USA. Electronic address: patterson.marc@mayo.edu. The gangliosidoses comprise a family of lysosomal storage diseases characterized by the accumulation of complex glycosphingolipids in the nervous system and other tissues, secondary to the deficient activity of lysosomal hydrolases or their associated activator proteins. GM1 and GM2 gangliosidosis are associated with deficiency of β-galactosidase and β-hexosaminidase respectively. All gangliosidoses are characterized by progressive neurodegeneration, the severity of which is proportional to the residual enzyme activity. The GM1 gangliosidoses are characterized by dysostosis, organomegaly and coarsening in their most severe forms, whereas children with classic infantile GM2 gangliosidosis (Tay-Sachs disease) are usually spared systemic involvement, except in the case of the Sandhoff variant, in which organomegaly may occur. Cherry-red macular spots occur in the early onset forms of the gangliosidoses, but are less frequently seen in the less severe, later onset phenotypes. Macrocephaly, an exaggerated startle response, cognitive decline, seizures, ataxia, and progressive muscular atrophy may occur in different forms of gangliosidosis. The diagnosis is made by assay of enzyme activity, and can be confirmed by mutation analysis. Carrier screening for Tay-Sachs disease has been remarkably successful in reducing the incidence of this disease in the at-risk Ashkenazi population. There are no proven disease-modifying therapies for the gangliosidoses. Copyright © 2013 Elsevier B.V. All rights reserved. DOI: 10.1016/B978-0-444-59565-2.00039-3 PMID: 23622392 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/17250509
1. Pediatr Int. 2007 Feb;49(1):70-5. doi: 10.1111/j.1442-200X.2007.02299.x. Cerebral hemiatrophy (Dyke-Davidoff-Masson syndrome) in childhood: clinicoradiological analysis of 19 cases. Atalar MH(1), Icagasioglu D, Tas F. Author information: (1)Department of Radiology, Division of Pediatric Neurology, Cumhuriyet University, Faculty of Medicine, Sivas, Turkey. mehmet5896@yahoo.com BACKGROUND: The purpose of this study was to emphasize the clinical and imaging findings of 19 child cases of cerebral hemiatrophy. METHODS: A total of 11 male and eight female patients underwent assessment with computed tomography and magnetic resonance imaging. The patients ranged from 1 to 17 years in age. The evaluated parameters were: location of the lesions, midline structural shift effect, ipsilateral calvarial and parenchymal changes. RESULTS: Left cerebral hemiatrophy was seen in 14 of the cases while right cerebral hemiatrophy was observed in five cases. Unilateral calvarial thickening was seen in 11 cases, hyperpneumatization of paranasal sinuses in five, and hypoplasia of the middle frontal cranial fossa in three patients. Cerebral peduncle atrophy was noted in seven cases. In total, 11 patients had thalamic atrophy and lentiform nucleus hypoplasia. In one case, cerebral hemiatrophy was associated with ipsilateral large schizencephalic cleft and absence of the septum pellucidum, whereas in another case, there was diffuse cerebellar atrophy associated with cerebral hemiatrophy. CONCLUSION: Computed tomography and, in particular, magnetic resonance imaging are the procedures of choice with respect to assessment of the etiology and extent of cerebral parenchymal involvement in cerebral hemiatrophy. DOI: 10.1111/j.1442-200X.2007.02299.x PMID: 17250509 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23672850
1. Ital J Pediatr. 2013 May 14;39:32. doi: 10.1186/1824-7288-39-32. Dyke-Davidoff-Masson syndrome: case report of fetal unilateral ventriculomegaly and hypoplastic left middle cerebral artery. Piro E(1), Piccione M, Marrone G, Giuffrè M, Corsello G. Author information: (1)Department of Sciences for Health Promotion and Mother and Child Care "Giuseppe D'Alessandro", University of Palermo, Palermo, Italy. ettore.piro@unipa.it Prenatal ultrasonographic detection of unilateral cerebral ventriculomegaly arises suspicion of pathological condition related to cerebrospinal fluid flow obstruction or cerebral parenchimal pathology. Dyke-Davidoff-Masson syndrome is a rare condition characterized by cerebral hemiatrophy, calvarial thickening, skull and facial asymmetry, contralateral hemiparesis, cognitive impairment and seizures. Congenital and acquired types are recognized and have been described, mainly in late childhood, adolescence and adult ages. We describe a female infant with prenatal diagnosis of unilateral left ventriculomegaly in which early brain MRI and contrast enhanced-MRI angiography, showed cerebral left hemiatrophy associated with reduced caliber of the left middle cerebral artery revealing the characteristic findings of the Dyke-Davidoff-Masson syndrome. Prenatal imaging, cerebral vascular anomaly responsible for the cerebral hemiatrophy and the early clinical evolution have never been described before in such a young child and complete the acquired clinical descriptions in older children. Differential diagnosis, genetic investigations, neurophysiologic assessments, short term clinical and developmental follow up are described. Dyke-Davidoff-Masson syndrome must be ruled out in differential diagnosis of fetal unilateral ventriculomegaly. Early clinical assessment, differential diagnosis and cerebral imaging including cerebral MRI angiography allow the clinicians to diagnose also in early infancy this rare condition. DOI: 10.1186/1824-7288-39-32 PMCID: PMC3666998 PMID: 23672850 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/1588015
1. J Child Neurol. 1992 Apr;7 Suppl:S41-50. doi: 10.1177/08830738920070010711. GM1 gangliosidosis type 2 in two siblings. Gascon GG(1), Ozand PT, Erwin RE. Author information: (1)Department of Pediatrics, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia. A sister and brother, now aged 7 and 9 years, presented with developmental arrest, gait disturbance, dementia, and a progressive myoclonic epilepsy syndrome with hyperacusis in the second year of life. Then, spastic quadriparesis led to a decerebrate state. In the absence of macular or retinal degeneration, organomegaly, and somatic-facial features suggesting mucopolysaccharidosis, the presence of hyperacusis together with sea-blue histiocytes in bone marrow biopsies and deficient beta-galactosidase activity but normal glucosidase, hexosaminidase, and neuraminidase activity on lysosomal enzyme assays constitutes the clinical-pathologic-biochemical profile of GM1 gangliosidosis type 2. This is a rare, late infantile onset, progressive gray-matter disease in which beta-galactosidase deficiency is largely localized to the brain, though it can be demonstrated in leukocytes and cultured skin fibroblasts. It must be distinguished from the Jansky-Bielschowsky presentation of neuronal ceroid lipofuscinosis, mitochondrial encephalopathy, lactic acidosis, strokelike episodes (MELAS) and myoclonic epilepsy with ragged-red fibers (MERRF) syndromes, atypical presentations of GM2 gangliosidoses (Tay-Sachs and Sandhoff's diseases), primary sialidosis (neuraminidase deficiency), galactosialidosis, and Alpers' disease. DOI: 10.1177/08830738920070010711 PMID: 1588015 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/3084261
1. Eur J Cell Biol. 1986 Mar;40(1):9-15. Immunoelectron microscopical localization of lysosomal beta-galactosidase and its precursor forms in normal and mutant human fibroblasts. Willemsen R, Hoogeveen AT, Sips HJ, van Dongen JM, Galjaard H. Immunoelectron microscopy was performed to study the biosynthesis of lysosomal beta-galactosidase (beta-gal) in normal and mutant human fibroblasts. Using polyclonal and monoclonal antibodies we show in normal cells precursor forms of beta-gal in the rough endoplasmic reticulum (RER) and in the Golgi apparatus throughout the stack of cisternae. In the lysosomes virtually all beta-gal exists as a high molecular weight multimer of mature enzyme. In the autosomal recessive disease GM1-gangliosidosis caused by a beta-gal deficiency and in galactosialidosis, associated with a combined deficiency of lysosomal neuraminidase and beta-gal, precursor forms of the latter enzyme are found in RER, Golgi and some labeling is present at the cell surface. The lysosomes remain unlabeled, indicative for the absence of enzyme molecules in this organelle. In galactosialidosis fibroblasts also no mature beta-gal is found in the lysosomes but in these cells the presence of the monomeric form can be increased by leupeptin (inhibition of proteolysis) whereas addition of a partly purified 32 kDa "protective protein" results in the restoration of high molecular weight beta-gal multimers in the lysosomes. PMID: 3084261 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/23189018
1. J Neurosci Rural Pract. 2012 Sep;3(3):411-3. doi: 10.4103/0976-3147.102646. Dyke-Davidoff-Masson syndrome. Behera MR(1), Patnaik S, Mohanty AK. Author information: (1)Department of Paediatrics, Kalinga Institute of Medical Sciences (KIMS), KIIT University, Patia, Bhubaneswar, Orissa, India. A 14-month-old male child presented with recurrent generalized seizures, spastic hemiplegia, microcephaly and had developmental delay in motor and speech domains. CT of the brain revealed characteristic features diagnostic of infantile type of cerebral hemiatrophy or Dyke-Davidoff-Masson syndrome. DOI: 10.4103/0976-3147.102646 PMCID: PMC3505357 PMID: 23189018 Conflict of interest statement: Conflict of Interest: None declared
http://www.ncbi.nlm.nih.gov/pubmed/19097769
1. Ann Anat. 2009 Apr;191(2):225-7. doi: 10.1016/j.aanat.2008.09.007. Epub 2008 Nov 13. Postmortal diagnosis of a Dyke-Davidoff-Masson syndrome in a 75-year-old woman---a case report. Stoevesandt D(1), Stock K, Spielmann RP, Heine HJ, Paulsen F, Bräuer L. Author information: (1)Department of Radiology, Martin Luther-University Halle-Wittenberg, 06120 Halle/Saale, Germany. dietrich.stoevesandt@medizin.uni-halle.de The Dyke-Davidoff-Masson syndrome is characterized by various symptoms related to hemiatrophy of the cerebrum and hypertrophy of the ipsilateral calvarium and paranasal sinuses. Clinical findings include hemiparesis or hemiplegia, seizures and/or mental retardation. The present report discusses the very unusual case of a late-diagnosed Dyke-Davidoff-Masson syndrome in a 75-year-old body donor who had suffered a left-sided stroke associated with the internal carotid artery in the course of tonsillitis at the age of 5. DOI: 10.1016/j.aanat.2008.09.007 PMID: 19097769 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/20236868
1. Epilepsy Behav. 2010 Apr;17(4):536-40. doi: 10.1016/j.yebeh.2010.02.006. Epub 2010 Mar 16. Right cerebral hemiatrophy: neurocognitive and electroclinical features. Demirtas-Tatlidede A(1), Yalcin AD, Uysal E, Forta H. Author information: (1)Department of Neurology, Sisli Etfal Research and Training Hospital, Istanbul, Turkey. aslidemirtas@yahoo.com The purpose of this study was to retrospectively evaluate the cognitive and electroclinical characteristics of right cerebral hemiatrophy (Dyke-Davidoff-Masson syndrome [DDMS]). Cognitive assessments with a particular emphasis on visuospatial functions, electroclinical features, and neuroimaging characteristics were analyzed for five patients with a clinically and neuroradiologically confirmed diagnosis of right-sided DDMS. Intelligence tests revealed mental retardation in all but one. Neuropsychological assessments demonstrated consistent impairments in tasks that have a spatial component (spatial processing and orientation discrimination), whereas attention, executive functions and verbal memory domains were variably impaired. Electroclinically, the main seizure types were simple partial motor, complex partial, and secondarily generalized seizures. Interictal EEG delineated lower amplitudes and slow background activity in the affected hemisphere. Overall, the cognitive performance of patients with DDMS encompasses a broad spectrum of impairments affecting multiple domains. Our findings support the concept that dorsal visual pathways responsible for spatial processing may be lateralized to the right hemisphere. Copyright (c) 2010 Elsevier Inc. All rights reserved. DOI: 10.1016/j.yebeh.2010.02.006 PMID: 20236868 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/15086521
1. J Neurochem. 2004 May;89(3):645-53. doi: 10.1046/j.1471-4159.2004.02381.x. N-butyldeoxygalactonojirimycin reduces neonatal brain ganglioside content in a mouse model of GM1 gangliosidosis. Kasperzyk JL(1), El-Abbadi MM, Hauser EC, D'Azzo A, Platt FM, Seyfried TN. Author information: (1)Department of Biology, Boston College, Chestnut Hill, Massachussetts, USA. GM1 gangliosidosis is a glycosphingolipid (GSL) lysosomal storage disease caused by a genetic deficiency of acid beta-galactosidase (beta-gal), the enzyme that catabolyzes GM1 within lysosomes. Accumulation of GM1 and its asialo form (GA1) occurs primarily in the brain, leading to progressive neurodegeneration and brain dysfunction. Substrate reduction therapy aims to decrease the rate of GSL biosynthesis to counterbalance the impaired rate of catabolism. The imino sugar N-butyldeoxygalactonojirimycin (NB-DGJ) is a competitive inhibitor of the ceramide-specific glucosyltransferase that catalyzes the first step in GSL biosynthesis. Neonatal C57BL/6J (B6) and beta-gal knockout (-/-) mice were injected daily from post-natal day 2 (p-2) to p-5 with either vehicle or NB-DGJ at 600 mg or 1200 mg/kg body weight. These drug concentrations significantly reduced total brain ganglioside and GM1 content in the B6 and the beta-gal (-/-) mice. Drug treatment had no significant effect on viability, body weight, brain weight, or brain water content in the B6 and beta-gal (-/-) mice. Significant elevations in neutral lipids (GA1, ceramide, and sphingomyelin) were observed in the NB-DGJ-treated beta-gal (-/-) mice, but were not associated with adverse effects. Also, NB-DGJ treatment of B6 and beta-gal (-/-) mice from p-2 to p-5 had no subsequent effect on brain ganglioside content at p-21. Our results show that NB-DGJ is effective in reducing total brain ganglioside and GM1 content at early neonatal ages. These findings suggest that substrate reduction therapy using NB-DGJ may be an effective early intervention for GM1 gangliosidosis and possibly other GSL lysosomal storage diseases. DOI: 10.1046/j.1471-4159.2004.02381.x PMID: 15086521 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/21559157
1. J Pediatr Neurosci. 2010 Jul;5(2):124-5. doi: 10.4103/1817-1745.76108. Dyke-Davidoff-Masson syndrome: Classical imaging findings. Singh P(1), Saggar K, Ahluwalia A. Author information: (1)Department of Radiodiagnosis, Dayanand Medical College & Hospital, Ludhiana, Punjab, India. A 15-year-old female presented with seizures, right-sided hemiparesis, hemiatrophy of the right side of the body and mental retardation. MRI brain revealed characteristic features diagnostic of congenital type of cerebral hemiatrophy or Dyke-Davidoff-Masson syndrome. DOI: 10.4103/1817-1745.76108 PMCID: PMC3087988 PMID: 21559157 Conflict of interest statement: Conflict of Interest: None declared.
http://www.ncbi.nlm.nih.gov/pubmed/23523468
1. Biochim Biophys Acta. 2013 Aug;1832(8):1194-206. doi: 10.1016/j.bbadis.2013.03.005. Epub 2013 Mar 20. Barth syndrome: cellular compensation of mitochondrial dysfunction and apoptosis inhibition due to changes in cardiolipin remodeling linked to tafazzin (TAZ) gene mutation. Gonzalvez F(1), D'Aurelio M, Boutant M, Moustapha A, Puech JP, Landes T, Arnauné-Pelloquin L, Vial G, Taleux N, Slomianny C, Wanders RJ, Houtkooper RH, Bellenguer P, Møller IM, Gottlieb E, Vaz FM, Manfredi G, Petit PX. Author information: (1)INSERM U-747 et Université Paris V-Descartes, Centre de Recherche des Saint-Pères, 45 Rue des Saint-Pères, 75006 Paris, France. Cardiolipin is a mitochondrion-specific phospholipid that stabilizes the assembly of respiratory chain complexes, favoring full-yield operation. It also mediates key steps in apoptosis. In Barth syndrome, an X chromosome-linked cardiomyopathy caused by tafazzin mutations, cardiolipins display acyl chain modifications and are present at abnormally low concentrations, whereas monolysocardiolipin accumulates. Using immortalized lymphoblasts from Barth syndrome patients, we showed that the production of abnormal cardiolipin led to mitochondrial alterations. Indeed, the lack of normal cardiolipin led to changes in electron transport chain stability, resulting in cellular defects. We found a destabilization of the supercomplex (respirasome) I+III2+IVn but also decreased amounts of individual complexes I and IV and supercomplexes I+III and III+IV. No changes were observed in the amounts of individual complex III and complex II. We also found decreased levels of complex V. This complex is not part of the supercomplex suggesting that cardiolipin is required not only for the association/stabilization of the complexes into supercomplexes but also for the modulation of the amount of individual respiratory chain complexes. However, these alterations were compensated by an increase in mitochondrial mass, as demonstrated by electron microscopy and measurements of citrate synthase activity. We suggest that this compensatory increase in mitochondrial content prevents a decrease in mitochondrial respiration and ATP synthesis in the cells. We also show, by extensive flow cytometry analysis, that the type II apoptosis pathway was blocked at the mitochondrial level and that the mitochondria of patients with Barth syndrome cannot bind active caspase-8. Signal transduction is thus blocked before any mitochondrial event can occur. Remarkably, basal levels of superoxide anion production were slightly higher in patients' cells than in control cells as previously evidenced via an increased protein carbonylation in the taz1Δ mutant in the yeast. This may be deleterious to cells in the long term. The consequences of mitochondrial dysfunction and alterations to apoptosis signal transduction are considered in light of the potential for the development of future treatments. Copyright © 2013 Elsevier B.V. All rights reserved. DOI: 10.1016/j.bbadis.2013.03.005 PMID: 23523468 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/24342716
1. Mol Genet Metab. 2014 Jan;111(1):26-32. doi: 10.1016/j.ymgme.2013.11.006. Epub 2013 Nov 19. Tafazzin splice variants and mutations in Barth syndrome. Kirwin SM(1), Manolakos A(2), Barnett SS(2), Gonzalez IL(2). Author information: (1)Molecular Diagnostics Laboratory, Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA. Electronic address: susan.kirwin@nemours.org. (2)Molecular Diagnostics Laboratory, Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, USA. Barth syndrome is caused by mutations in the TAZ (tafazzin) gene on human chromosome Xq28. The human tafazzin gene produces four major mRNA splice variants; two of which have been shown to be functional (TAZ lacking exon 5 and full-length) in complementation studies with yeast and Drosophila. This study characterizes the multiple alternative splice variants of TAZ mRNA and their proportions in blood samples from a cohort of individuals with Barth syndrome (BTHS). Because it has been reported that collection and processing methods can affect the expression of various genes, we tested and chose a stabilizing medium for collecting, shipping and processing of the blood samples of these individuals. In both healthy controls and in BTHS individuals, we found a greater variety of alternatively spliced forms than previously described, with a sizeable proportion of minor splice variants besides the four dominant isoforms. Individuals with certain exonic and intronic splice mutations produce additional mutant mRNAs that could be translated into two or more proteins with different amino acid substitutions in a single individual. A fraction of the minor splice variants is predicted to be non-productive. Copyright © 2013 Elsevier Inc. All rights reserved. DOI: 10.1016/j.ymgme.2013.11.006 PMID: 24342716 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/24481314
1. Aging (Albany NY). 2014 Jan;6(1):48-57. doi: 10.18632/aging.100633. BMAL1-dependent regulation of the mTOR signaling pathway delays aging. Khapre RV(1), Kondratova AA, Patel S, Dubrovsky Y, Wrobel M, Antoch MP, Kondratov RV. Author information: (1)Center for Gene Regulation in Health and Diseases, BGES, Cleveland State University, Cleveland, OH. The circadian clock, an internal time-keeping system, has been linked with control of aging, but molecular mechanisms of regulation are not known. BMAL1 is a transcriptional factor and core component of the circadian clock; BMAL1 deficiency is associated with premature aging and reduced lifespan. Here we report that activity of mammalian Target of Rapamycin Complex 1 (mTORC1) is increased upon BMAL1 deficiency both in vivo and in cell culture. Increased mTOR signaling is associated with accelerated aging; in accordance with that, treatment with the mTORC1 inhibitor rapamycin increased lifespan of Bmal1-/- mice by 50%. Our data suggest that BMAL1 is a negative regulator of mTORC1 signaling. We propose that the circadian clock controls the activity of the mTOR pathway through BMAL1-dependent mechanisms and this regulation is important for control of aging and metabolism. DOI: 10.18632/aging.100633 PMCID: PMC3927809 PMID: 24481314 [Indexed for MEDLINE] Conflict of interest statement: The authors of this paper declare no conflict of interests.
http://www.ncbi.nlm.nih.gov/pubmed/20519775
1. Aging (Albany NY). 2010 May;2(5):285-97. doi: 10.18632/aging.100142. Circadian clock proteins control adaptation to novel environment and memory formation. Kondratova AA(1), Dubrovsky YV, Antoch MP, Kondratov RV. Author information: (1)Lerner Research Institute, Cleveland Clinic, OH 44195, USA. Comment in Aging (Albany NY). 2010 May;2(5):251-4. doi: 10.18632/aging.100144. Aging (Albany NY). 2010 May;2(5):259-60. doi: 10.18632/aging.100148. Aging (Albany NY). 2010 Jun;2(6):320-1. doi: 10.18632/aging.100154. Deficiency of the transcription factor BMAL1, a core component of the circadian clock, results in an accelerated aging phenotype in mice. The circadian clock regulates many physiological processes and was recently implicated in control of brain-based activities, such as memory formation and the regulation of emotions. Aging is accompanied by the decline in brain physiology, particularly decline in the response and adaptation to novelty. We investigated the role of the circadian clock in exploratory behavior and habituation to novelty using the open field paradigm. We found that mice with a deficiency of the circadian transcription factor BMAL1 display hyperactivity in novel environments and impaired intra- and intersession habituation, indicative of defects in short- and long-term memory formation. In contrast, mice double-deficient for the circadian proteins CRY1 and CRY2 (repressors of the BMAL1-mediated transcription) demonstrate reduced activity and accelerated habituation when compared to wild type mice. Mice with mutation in theClock gene (encoding the BMAL1 transcription partner) show normal locomotion, but increased rearing activity and impaired intersession habituation. BMAL1 is highly expressed in the neurons of the hippocampus - a brain region associated with spatial memory formation; BMAL1 deficiency disrupts circadian oscillation in gene expression and reactive oxygen species homeostasis in the brain, which may be among the possible mechanisms involved. Thus, we suggest that the BMAL1:CLOCK activity is critical for the proper exploratory and habituation behavior, and that the circadian clock prepares organism for a new round of everyday activities through optimization of behavioral learning. DOI: 10.18632/aging.100142 PMCID: PMC2898019 PMID: 20519775 [Indexed for MEDLINE] Conflict of interest statement: The authors of this manuscript have no conflict of interests to declare.
http://www.ncbi.nlm.nih.gov/pubmed/21149897
1. Aging (Albany NY). 2010 Dec;2(12):936-44. doi: 10.18632/aging.100241. Deficiency of circadian protein CLOCK reduces lifespan and increases age-related cataract development in mice. Dubrovsky YV(1), Samsa WE, Kondratov RV. Author information: (1)Department of Biological, Geological and Environmental Sciences and Center for Gene Regulation in Health and Disease, Cleveland State University, Cleveland, OH 44115, USA. Circadian clock is implicated in the regulation of aging. The transcription factor CLOCK, a core component of the circadian system, operates in complex with another circadian clock protein BMAL1. Recently it was demonstrated that BMAL1 deficiency results in premature aging in mice. Here we investigate the aging of mice deficient for CLOCK protein. Deficiency of the CLOCK protein significantly affects longevity: the average lifespan of Clock-/- mice is reduced by 15% compared with wild type mice, while maximum lifespan is reduced by more than 20%. CLOCK deficiency also results in the development of two age-specific pathologies in these mice, cataracts and dermatitis, at a much higher rate than in wild type mice. In contrast to BMAL1 deficient animals, Clock-/- mice do not develop a premature aging phenotype and do not develop the multiple age-associated pathologies characteristic of BMAL1 deficiency. Thus, although CLOCK and BMAL1 form a transcriptional complex, the physiological result of their deficiency is different. Our results suggest that CLOCK plays an important role in aging, specifically; CLOCK activity is critical for the regulation of normal physiology and aging of the lens and skin. DOI: 10.18632/aging.100241 PMCID: PMC3034182 PMID: 21149897 [Indexed for MEDLINE] Conflict of interest statement: The authors of this paper declare no conflict of interests.
http://www.ncbi.nlm.nih.gov/pubmed/23104054
1. Nat Struct Mol Biol. 2012 Dec;19(12):1257-65. doi: 10.1038/nsmb.2434. Epub 2012 Oct 28. Phf19 links methylated Lys36 of histone H3 to regulation of Polycomb activity. Ballaré C(1), Lange M, Lapinaite A, Martin GM, Morey L, Pascual G, Liefke R, Simon B, Shi Y, Gozani O, Carlomagno T, Benitah SA, Di Croce L. Author information: (1)Department of Gene Regulation and Stem Cells, Centre for Genomic Regulation (CRG), Barcelona, Spain. Comment in Nat Struct Mol Biol. 2012 Dec;19(12):1214-5. doi: 10.1038/nsmb.2458. Polycomb-group proteins are transcriptional repressors with essential roles in embryonic development. Polycomb repressive complex 2 (PRC2) contains the methyltransferase activity for Lys27. However, the role of other histone modifications in regulating PRC2 activity is just beginning to be understood. Here we show that direct recognition of methylated histone H3 Lys36 (H3K36me), a mark associated with activation, by the PRC2 subunit Phf19 is required for the full enzymatic activity of the PRC2 complex. Using NMR spectroscopy, we provide structural evidence for this interaction. Furthermore, we show that Phf19 binds to a subset of PRC2 targets in mouse embryonic stem cells and that this is required for their repression and for H3K27me3 deposition. These findings show that the interaction of Phf19 with H3K36me2 and H3K36me3 is essential for PRC2 complex activity and for proper regulation of gene repression in embryonic stem cells. DOI: 10.1038/nsmb.2434 PMCID: PMC3926938 PMID: 23104054 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/22101268
1. Cell Cycle. 2011 Dec 1;10(23):4162-9. doi: 10.4161/cc.10.23.18381. Epub 2011 Dec 1. Circadian clock protein BMAL1 regulates cellular senescence in vivo. Khapre RV(1), Kondratova AA, Susova O, Kondratov RV. Author information: (1)Center for Gene Regulation in Health and Disease, BGES Department, Cleveland State University, Cleveland, OH, USA. Comment in Cell Cycle. 2012 Jan 15;11(2):213-4. doi: 10.4161/cc.11.2.18786. Deficiency of the circadian clock transcriptional factor BMAL1 results in the development of premature aging in mice. In agreement with the accelerated aging phenotype, we observed an increase in the number of senescent cells in different tissues (lungs, liver and spleen) of Bmal1(-/-) mice, which suggests the important role of BMAL1 in the control of senescence in vivo. However, no difference in the rate of proliferation and senescence between primary fibroblasts isolated from wild-type and Bmal1(-/-) mice has been detected, suggesting that BMAL1 does not play a significant role in replicative senescence in vitro. BMAL1 deficient fibroblasts had an increased sensitivity to hydrogen peroxide treatment, and reduced sensitivity to DNA damaging anticancer drugs etoposide and daunorubicin. Increased sensitivity of Bmal1(-/-) cells to oxidative stress was p53 independent and correlated with the disrupted regulation of reactive oxygen species (ROS) homeostasis in BMAL1 deficient cells: indeed, circadian oscillations of ROS level can be induced in wild-type but not in Bmal1(-/-) cells. We propose that BMAL1 is important for the regulation of oxidative stress and DNA damage responses, while deregulation of these processes upon BMAL1 deficiency leads to development of stress induced senescence in vivo. DOI: 10.4161/cc.10.23.18381 PMCID: PMC3272294 PMID: 22101268 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/24801725
1. Cardiovasc Hematol Disord Drug Targets. 2014;14(2):98-106. doi: 10.2174/1871529x14666140505123753. Cardiolipin metabolism and the role it plays in heart failure and mitochondrial supercomplex formation. Mejia EM, Cole LK, Hatch GM(1). Author information: (1)Department of Pharmacology & Therapeutics, University of Manitoba, Manitoba Institute of Child Health, 501C-715 McDermot Avenue, Winnipeg, Manitoba, Canada, R3E 3P4. ghatch@mich.ca. Cardiolipin is a major membrane phospholipid in the mitochondria and is essential for cellular energy metabolism mediated through mitochondrial oxidative phosphorylation. Recent studies indicate that it plays a diverse role in cellular metabolism. Eukaryotic cardiolipin is synthesized de novo from phosphatidic acid via the cytidine-5'-diphosphate-1,2-diacyl-sn-glycerol pathway and is deacylated to monolysocardiolipin in order for it to be remodelled into the form that is observed in mitochondrial membranes. This resynthesis of deacylated cardiolipin from monolysocardiolipin occurs via the Barth Syndrome gene product tafazzin and acyllysocardiolipin acyltransferase-1, monolysocardiolipin acyltransferase-1 and the alpha subunit of trifunctional protein. Heart failure is a disease condition in which the amount and type of cardiolipin is altered. Several animal models have been generated to study the role of altered cardiolipin in heart failure. In many of these models loss of the tetralinoleoyl-cardiolipin species is observed during the development of the heart failure. In the doxycycline inducible short hairpin RNA tafazzin knock down mouse, loss of tetralinoleoyl-cardiolipin is associated with a mitochondrial bioenergetic disruption. Reduction in mitochondrial supercomplex formation and NADH dehydrogenase activity within these supercomplexes is observed. Modulation of CL fatty acyl composition may serve as a therapeutic strategy for the treatment of several pathologies including cardiac dysfunction.We propose that increasing cardiolipin may improve mitochondrial function and potentially serve as a therapy for diseases which exhibit mitochondrial dysfunction involving reduced cardiolipin. DOI: 10.2174/1871529x14666140505123753 PMID: 24801725 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/24093814
1. Prague Med Rep. 2013;114(3):139-53. doi: 10.14712/23362936.2014.16. Novel mutations in the TAZ gene in patients with Barth syndrome. Mazurová S(1), Tesařová M, Magner M, Houšťková H, Hansíková H, Augustínová J, Tomek V, Vondráčková A, Zeman J, Honzík T. Author information: (1)Department of Pediatrics and Adolescent Medicine, First Faculty of Medicine, Charles University in Prague and General University Hospital in Prague, Prague, Czech Republic. Barth syndrome is an X-linked recessive disorder that is caused by mutations in Taffazin gene (TAZ), leading to severe cardiolipin deficiency which results in respiratory chain dysfunction. Barth syndrome is characterized by cardiomyopathy, neutropenia, skeletal myopathy, growth deficiency and 3-methylglutaconic aciduria. In this paper, we present clinical, biochemical and molecular data of the first four Czech patients from four unrelated families diagnosed with this rare disease. The mean age of onset was 5.5 ± 3.8 months. One child suffered from sudden cardiac death at the age of 2 years, the age of living patients is between 3 and 13 years. Muscle hypotonia was present in all four patients; cardiomyopathy and growth retardation in three and neutropenia in two of them. Two patients manifested a dilated and one patient a hypertrophic cardiomyopathy. A characteristic laboratory abnormality was the intermittently increased excretion of 3-methylglutaconic acid. Three novel hemizygous mutations in the TAZ gene were found (c.584G>T; c.109+6T>C; c.86G>A). We conclude that Barth syndrome should be included in differential diagnosis of cardiomyopathy in childhood, especially in the cooccurrence of dilated cardiomyopathy and 3-methylglutaconic aciduria. DOI: 10.14712/23362936.2014.16 PMID: 24093814 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/24977128
1. J Epilepsy Res. 2014 Jun 30;4(1):24-7. doi: 10.14581/jer.14006. eCollection 2014 Jun. Dyke-davidoff-masson syndrome: cases of two brothers and literature review. Park KI(1), Chung JM(1), Kim JY(1). Author information: (1)Department of Neurology, Inje University College of Medicine, Seoul Paik Hospital, Seoul, Korea. Dyke-Davidoff-Masson syndrome (DDMS) has cerebral hemiatrophy and compensatory ipsilateral skull thickening, and is manifested by recurrent seizures and hemiparesis. We present one case with typical DDMS, who had a brother suffering from epilepsy with mild imaging abnormality relevant to DDMS and similar seizure semiology. A 26-year-old man had a history of developmental delay, mental retardation, hemiparesis and recurrent seizures. His brother, 23-year-old man had also experienced recurrent seizures, but he had no neurological deficits. Older brother experienced focal motor seizures with/without secondary generalization. Sometimes, he noted an auditory aura. MRI demonstrated the hemispheric atrophy with the adjacent bony hypertrophy. The seizures of younger brother were mainly of the auditory type and the MRI showed mild hemispheric atrophy with hippocampal sclerosis without any bony change. Our sibling cases might have a familial predisposition and support the idea that clinical courses and radiological findings of DDMS are varied even within one family. DOI: 10.14581/jer.14006 PMCID: PMC4066624 PMID: 24977128
http://www.ncbi.nlm.nih.gov/pubmed/22023389
1. Eur J Haematol. 2012 Mar;88(3):195-209. doi: 10.1111/j.1600-0609.2011.01725.x. Epub 2011 Dec 4. The cellular and molecular mechanisms for neutropenia in Barth syndrome. Makaryan V(1), Kulik W, Vaz FM, Allen C, Dror Y, Dale DC, Aprikyan AA. Author information: (1)Department of Medicine, University of Washington, Seattle, WA, USA. Barth syndrome (BTHS), a rare, X-linked, recessive disease, is characterized by neutropenia and cardiomyopathy. BTHS is caused by loss-of-function mutations of the tafazzin (TAZ) gene. We developed a model of BTHS by transfecting human HL60 myeloid progenitor cells with TAZ-specific shRNAs. Results demonstrate a significant downregulation in TAZ expression, mimicking the effects of naturally occurring truncation mutations in TAZ. Flow cytometry analyses of cells with TAZ-specific, but not scrambled, shRNAs demonstrate nearly twofold increase in the proportion of annexin V-positive cells and significantly increased dissipation of mitochondrial membrane potential as determined by DIOC6 staining. Transfection of TAZ-specific shRNA had similar effects in U937 myeloid cells but not in lymphoid cell lines. Further studies in HL60 myeloid progenitor cells revealed aberrant release of cytochrome c from mitochondria and significantly elevated levels of activated caspase-3 in response to TAZ knockdown. Treatment with caspase-specific inhibitor zVAD-fmk resulted in substantially reduced apoptosis to near-normal levels. These data suggest that neutropenia in BTHS is attributable to increased dissipation of mitochondrial membrane potential, aberrant release of cytochrome c, activation of caspase-3, and accelerated apoptosis of myeloid progenitor cells, and that this defect can be partially restored in vitro by treatment with caspase-specific inhibitors. © 2011 John Wiley & Sons A/S. DOI: 10.1111/j.1600-0609.2011.01725.x PMCID: PMC4445723 PMID: 22023389 [Indexed for MEDLINE]
http://www.ncbi.nlm.nih.gov/pubmed/25941633
1. Meta Gene. 2015 Apr 22;4:92-106. doi: 10.1016/j.mgene.2015.04.001. eCollection 2015 Jun. Structural and functional analyses of Barth syndrome-causing mutations and alternative splicing in the tafazzin acyltransferase domain. Hijikata A(1), Yura K(2), Ohara O(3), Go M(4). Author information: (1)Nagahama Institute of Bio-Science and Technology, 1266 Tamura-cho, Nagahama, Shiga 526-0829, Japan. (2)Graduate School of Humanities and Sciences, Ochanomizu University, 2-1-1 Otsuka, Bunkyo-ku, Tokyo 112-8610, Japan. (3)Laboratory for Integrative Genomics, RIKEN Center for Integrative Medical Sciences, 1-7-22 Suehiro-cho, Tsurumi-ku, Yokohama, Kanagawa 230-0045, Japan ; Department of Technology Development, Kazusa DNA Research Institute, 2-6-7 Kazusa-Kamatari, Kisarazu, Chiba 292-0818, Japan. (4)Nagahama Institute of Bio-Science and Technology, 1266 Tamura-cho, Nagahama, Shiga 526-0829, Japan ; Research Organization of Information and Systems, 4-3-13, Toranomon, Minatoku, Tokyo 105-0001, Japan. Tafazzin is a mitochondrial phospholipid transacylase, and its mutations cause Barth syndrome (BTHS). Human tafazzin gene produces four distinct alternatively spliced transcripts. To understand the molecular mechanisms of tafazzin deficiency, we performed an atomic resolution analysis of the influence of the BTHS mutations and of alternative splicing on the structure and function of tafazzin. From the three-dimensional (3D) homology modeling of tafazzin, we identified candidate amino acid residues that contribute to cardiolipin binding and to mitochondrial membrane associations that facilitate acyl-transfer reactions. Primate specific exon 5, which is alternatively spliced, is predicted to correspond to an intrinsically unstructured region in the protein. We proposed that this region should change the substrate-binding affinity and/or contribute to primate-specific molecular interactions. Exon 7, another alternatively spliced exon, encodes a region forming a part of the putative substrate-binding cleft, suggesting that the gene products lacking exon 7 will lose their substrate-binding ability. We demonstrate a clear localization of the BTHS mutations at residues responsible for membrane association, substrate binding, and the conformational stability of tafazzin. These findings provide new insights into the function of defective tafazzin and the pathogenesis of BTHS at the level of protein 3D structure and the evolution of alternatively spliced exons in primates. DOI: 10.1016/j.mgene.2015.04.001 PMCID: PMC4412953 PMID: 25941633