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

• Prif Awduron: Linde F. Bouwman (Awdur), Bianca den Hamer (Awdur), Anita van den Heuvel (Awdur), Marnix Franken (Awdur), Michaela Jackson (Awdur), Chrissa A. Dwyer (Awdur), Stephen J. Tapscott (Awdur), Frank Rigo (Awdur), Silvère M. van der Maarel (Awdur), Jessica C. de Greef (Awdur)
• Fformat: Llyfr
• Cyhoeddwyd: Elsevier, 2021-12-01T00:00:00Z.
• Pynciau: article
• Mynediad Ar-lein: Connect to this object online.

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...