Open AccessThe prospects of targeting DUX4 in facioscapulohumeral muscular dystrophy
Author(s) -
Linde F. Bouwman,
Silvère M. van der Maarel,
Jessica C. de Greef
Publication year - 2020
Publication title -
current opinion in neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 119
eISSN - 1473-6551
pISSN - 1350-7540
DOI - 10.1097/wco.0000000000000849
Subject(s) - facioscapulohumeral muscular dystrophy , psychological repression , in vivo , transcription factor , muscular dystrophy , gene , biology , cancer research , bioinformatics , medicine , genetics , gene expression
Facioscapulohumeral muscular dystrophy (FSHD) is a neuromuscular disorder, which is caused by incomplete repression of the transcription factor double homeobox 4 (DUX4) in skeletal muscle. To date, there is no DUX4-targeting treatment to prevent or delay disease progression. In the present review, we summarize developments in therapeutic strategies with the focus on inhibiting DUX4 and DUX4 target gene expression.