BET bromodomain inhibitors and agonists of the beta-2 adrenergic receptor identified in screens for compounds that inhibit DUX4 expression in FSHD muscle cells.

Amy E CampbellJonathan OlivaMatthew P YatesJun Wen ZhongSean C ShadleLauren SniderNikita SinghShannon TaiYosuke HiramukiRabi TawilSilvère M van der MaarelStephen J TapscottFrancis M Sverdrup

Published in: Skeletal muscle (2017)

These data uncover pathways involved in the regulation of DUX4 expression in somatic cells, provide potential candidate classes of compounds for FSHD therapeutic development, and create an important opportunity for mechanistic studies that may uncover additional therapeutic targets.

Keyphrases

induced apoptosis
poor prognosis
cell cycle arrest
binding protein
endoplasmic reticulum stress
skeletal muscle
oxidative stress
cell death
signaling pathway
high throughput
electronic health record
gene expression
dna methylation
big data