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

• Үндсэн зохиолчид: Linde F. Bouwman (Зохиогч), Bianca den Hamer (Зохиогч), Anita van den Heuvel (Зохиогч), Marnix Franken (Зохиогч), Michaela Jackson (Зохиогч), Chrissa A. Dwyer (Зохиогч), Stephen J. Tapscott (Зохиогч), Frank Rigo (Зохиогч), Silvère M. van der Maarel (Зохиогч), Jessica C. de Greef (Зохиогч)
• Формат: Ном
• Хэвлэсэн: Elsevier, 2021-12-01T00:00:00Z.
• Нөхцлүүд: article
• Онлайн хандалт: 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...