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BETi Compounds Alter DUX4 Expression

Posted by George Shaw on September 22, 2017

Fran Sverdrup, Phd - Research Fellow, Center for World Health and Medicine, Saint Louis University

Fran Sverdrup, Phd - Research Fellow, Center for World Health and Medicine, Saint Louis University

The recent paper from Amy Campbell and Fran Sverdrup (see also our Ask the Expert article wherein Fran discusses his work on BET inhibitors) provides details on BETi compounds that appear to suppress DUX4 expression. This not only provides another potential route to therapy, but also is one more clue to the needed mechanistic understanding of the process. The group "revealed unanticipated roles for BET proteins and beta-2 adrenergic receptor signaling in the processes controlling DUX4 expression."

Fran Sverdrup's entry into the world of FSHD research is in part documented in this St Louis Magazine article from July 2016. Fran has been relentless in his dedication and drive In Pursuit of a Cure, and attended our 2016 Portland Summit. Fran's lab was also one of the stops on the C2C4 FSHD Cross-Country Bicycling Journey, starting here in Seattle with Friends of FSH.

Publication: BET bromodomain inhibitors and agonists of the beta-2 adrenergic receptor identified in screens for compounds that inhibit DUX4 expression in FSHD muscle cells
Published 4-Sep-2017