RNA Interference Inhibits DUX4-induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy

No treatment exists for facioscapulohumeral muscular dystrophy (FSHD), one of the most common inherited muscle diseases. Although FSHD can be debilitating, little effort has been made to develop targeted therapies. This lack of focus on targeted FSHD therapy perpetuated because the genes and pathway...

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Autors principals: Wallace, Lindsay M, Liu, Jian, Domire, Jacqueline S, Garwick-Coppens, Sara E, Guckes, Susan M, Mendell, Jerry R, Flanigan, Kevin M, Harper, Scott Q.
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2012
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3392971/
https://ncbi.nlm.nih.gov/pubmed/22508491
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.68
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