RNAi-mediated Gene Silencing of Mutant Myotilin Improves Myopathy in LGMD1A Mice

• المؤلفون الرئيسيون: Jian Liu (مؤلف), Lindsay M Wallace (مؤلف), Sara E Garwick-Coppens (مؤلف), Darcée D Sloboda (مؤلف), Carol S Davis (مؤلف), Chady H Hakim (مؤلف), Michael A Hauser (مؤلف), Susan V Brooks (مؤلف), Jerry R Mendell (مؤلف), Scott Q Harper (مؤلف)
• التنسيق: كتاب
• منشور في: Elsevier, 2014-01-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: Connect to this object online.

Recent progress suggests gene therapy may one day be an option for treating some forms of limb girdle muscular dystrophy (LGMD). Nevertheless, approaches targeting LGMD have so far focused on gene replacement strategies for recessive forms of the disease. In contrast, no attempts have been made to d...