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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 DUX4 expression have the potential to treat FSHD. Apabetalone is...

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Detalles Bibliográficos
Autores principales: Sarsons, Christopher D., Gilham, Dean, Tsujikawa, Laura M., Wasiak, Sylwia, Fu, Li, Rakai, Brooke D., Stotz, Stephanie C., Carestia, Agostina, Sweeney, Michael, Kulikowski, Ewelina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604783/
https://www.ncbi.nlm.nih.gov/pubmed/37893058
http://dx.doi.org/10.3390/biomedicines11102683