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

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...

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Main Authors: Liu, Jian, Wallace, Lindsay M, Garwick-Coppens, Sara E, Sloboda, Darcée D, Davis, Carol S, Hakim, Chady H, Hauser, Michael A, Brooks, Susan V, Mendell, Jerry R, Harper, Scott Q
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2014
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Original Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4013433/
https://ncbi.nlm.nih.gov/pubmed/24781192
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2014.13
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