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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Detalhes bibliográficos

Principais autores:	Linde F. Bouwman, Bianca den Hamer, Anita van den Heuvel, Marnix Franken, Michaela Jackson, Chrissa A. Dwyer, Stephen J. Tapscott, Frank Rigo, Silvère M. van der Maarel, Jessica C. de Greef
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2021-12-01
coleção:	Molecular Therapy: Nucleic Acids
Assuntos:	facioscapulohumeral muscular dystrophy DUX4 therapy antisense oligonucleotide ACTA1-MCM FLExDUX4 mouse model
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2162253121002390
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