کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
3080966 | 1189361 | 2010 | 7 صفحه PDF | دانلود رایگان |
عنوان انگلیسی مقاله ISI
Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials
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موضوعات مرتبط
علوم زیستی و بیوفناوری
علم عصب شناسی
علوم اعصاب تکاملی
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چکیده انگلیسی
Duchenne muscular dystrophy (DMD) is characterised by the absence of dystrophin in muscle biopsies, although residual dystrophin can be present, either as dystrophin-positive (revertant) fibres or traces. As restoration of dystrophin expression is the end point of clinical trials, such residual dystrophin is a key factor in recruitment of patients and may also confound the analysis of dystrophin restoration in treated patients, if, as previously observed in the mdx mouse, revertant fibres increase with age. In 62% of the diagnostic biopsies reports of 65 DMD patients studied, traces or revertants were recorded with no correlation between traces or revertants, the patients' performance, or corticosteroids response. In nine of these patients, there was no increase in traces or revertants in biopsies taken a mean of 8.23Â years (5.8-10.4Â years) after the original diagnostic biopsy. This information should help in the design and execution of clinical trials focused on dystrophin restoration strategies.
ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Neuromuscular Disorders - Volume 20, Issue 5, May 2010, Pages 295-301
Journal: Neuromuscular Disorders - Volume 20, Issue 5, May 2010, Pages 295-301
نویسندگان
Virginia Arechavala-Gomeza, Maria Kinali, Lucy Feng, Michela Guglieri, Geraldine Edge, Marion Main, David Hunt, Jan Lehovsky, Volker Straub, Kate Bushby, Caroline A. Sewry, Jennifer E. Morgan, Francesco Muntoni,