Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics

Derepression of DUX4 in skeletal muscle has emerged as a likely cause of pathology in facioscapulohumeral muscular dystrophy (FSHD). Here we report on the use of antisense phosphorodiamidate morpholino oligonucleotides to suppress DUX4 expression and function in FSHD myotubes and xenografts. The mos...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Chen, Jennifer CJ, King, Oliver D, Zhang, Yuanfan, Clayton, Nicholas P, Spencer, Carrie, Wentworth, Bruce M, Emerson, Charles P, Wagner, Kathryn R
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5023379/
https://ncbi.nlm.nih.gov/pubmed/27378237
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.111
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