Apabetalone, a Clinical-Stage, Selective BET Inhibitor, Opposes DUX4 Target Gene Expression in Primary Human FSHD Muscle Cells

Facioscapulohumeral dystrophy (FSHD) is a muscle disease caused by inappropriate expression of the double homeobox 4 (DUX4) gene in skeletal muscle, and its downstream activation of pro-apoptotic transcriptional programs. Inhibitors of <i>DUX4</i> expression have the potential to treat FSHD. Apabeta...

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Autors principals:	Christopher D. Sarsons, Dean Gilham, Laura M. Tsujikawa, Sylwia Wasiak, Li Fu, Brooke D. Rakai, Stephanie C. Stotz, Agostina Carestia, Michael Sweeney, Ewelina Kulikowski
Format:	Artigo
Idioma:	Inglês
Publicat:	MDPI AG 2023-09-01
Col·lecció:	Biomedicines
Matèries:	facioscapulohumeral muscular dystrophy FSHD DUX4 bromodomain and extra-terminal domain BET inhibitor transcriptome
Accés en línia:	https://www.mdpi.com/2227-9059/11/10/2683
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