Sverdrup Lab Uncovers Pathways to Treatment of FSHD

Francis Sverdrup, Ph.D., has identified several potential new drug therapies for the treatment of facioscapulohumeral muscualr dystrophy (FSHD). His findings were published in Skeletal Muscle.

FSHD is an inherited disease affecting mainly young adults. It is caused by mis-expression of the double homeobox 4 (DUX4) transcription factor in skeletal muscle. Sverdrup and colleagues screened known drug libraries and tested drugs on FSHD cells to identify the pathways involved in expression of the DUX4 gene, providing potential new therapies for treatment of the disease.

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Sverdrup Skel Musc Fig 7 2017

Model of DUX4 regulation by BET proteins and beta-2 adrenergic signaling.

Department of Biochemistry and Molecular Biology