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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Detalles Bibliográficos
Autores principales: Chen, Jennifer CJ, King, Oliver D, Zhang, Yuanfan, Clayton, Nicholas P, Spencer, Carrie, Wentworth, Bruce M, Emerson, Charles P, Wagner, Kathryn R
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5023379/
https://www.ncbi.nlm.nih.gov/pubmed/27378237
http://dx.doi.org/10.1038/mt.2016.111

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5023379/
https://www.ncbi.nlm.nih.gov/pubmed/27378237
http://dx.doi.org/10.1038/mt.2016.111

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