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

• Main Authors: Jian Liu (Author), Lindsay M Wallace (Author), Sara E Garwick-Coppens (Author), Darcée D Sloboda (Author), Carol S Davis (Author), Chady H Hakim (Author), Michael A Hauser (Author), Susan V Brooks (Author), Jerry R Mendell (Author), Scott Q Harper (Author)
• Format: Book
• Published: Elsevier, 2014-01-01T00:00:00Z.
• Subjects: article
• Online Access: 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...