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Therapeutic Strategy and Clinical Path of Facioscapulohumeral Muscular Dystrophy: Review of the Current Literature

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant genetic disease, which is caused by the mistaken expression of double homeobox protein 4 protein 4 (DUX4) in skeletal muscle. Patients with FSHD are usually accompanied by degenerative changes in the face, shoulders, and upper mu...

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Principais autores: Qi Xie, Guangmei Ma, Yafeng Song
פורמט: Artigo
שפה:Inglês
יצא לאור: MDPI AG 2024-09-01
סדרה:Applied Sciences
נושאים:
facioscapulohumeral muscular dystrophy
genetic treatment
oligonucleotides
Crispr
DUX4
גישה מקוונת:https://www.mdpi.com/2076-3417/14/18/8222
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