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Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most prevalent skeletal muscle dystrophies. Skeletal muscle pathology in individuals with FSHD is caused by inappropriate expression of the transcription factor DUX4, which activates different myotoxic pathways. At the moment there is no mo...

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Detalles Bibliográficos
Autores principales:	Bouwman, Linde F., den Hamer, Bianca, van den Heuvel, Anita, Franken, Marnix, Jackson, Michaela, Dwyer, Chrissa A., Tapscott, Stephen J., Rigo, Frank, van der Maarel, Silvère M., de Greef, Jessica C.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8526479/ https://www.ncbi.nlm.nih.gov/pubmed/34729250 http://dx.doi.org/10.1016/j.omtn.2021.09.010

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