کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
8841090 1614705 2018 60 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
Experimental and Clinical Strategies for Treating Spinocerebellar Ataxia Type 3
موضوعات مرتبط
علوم زیستی و بیوفناوری علم عصب شناسی علوم اعصاب (عمومی)
پیش نمایش صفحه اول مقاله
Experimental and Clinical Strategies for Treating Spinocerebellar Ataxia Type 3
چکیده انگلیسی
Spinocerebellar ataxia type 3 (SCA3), or Machado-Joseph disease (MJD), is an autosomal dominant neurodegenerative disorder caused by the expansion of a polyglutamine (polyQ) tract in the ataxin-3 protein. To date, there is no effective therapy available to prevent progression of this disease. However, clinical strategies for alleviating various symptoms are imperative to promote a better quality of life for SCA3/MJD patients. Furthermore, experimental therapeutic strategies, including gene silencing or mutant protein clearance, mutant polyQ protein modification, stabilizing the native protein conformation, rescue of cellular dysfunction and neuromodulation to slow the progression of SCA3/MJD, have been developed. In this study, based on the current knowledge, I detail the clinical and experimental therapeutic strategies for treating SCA3/MJD, paying particular attention to drug discovery.
ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: Neuroscience - Volume 371, 10 February 2018, Pages 138-154
نویسندگان
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