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

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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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Elsevier 2021-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
facioscapulohumeral muscular dystrophy
DUX4
therapy
antisense oligonucleotide
ACTA1-MCM
FLExDUX4 mouse model
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253121002390

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http://www.sciencedirect.com/science/article/pii/S2162253121002390

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