Peer-Reviewed Publication
Sci Rep2026June 16, 2026Journal Article
Identification of compounds that repress DUX4 expression in facioscapulohumeral muscular dystrophy.
Ning Chang1, Hannah P Moore1,2, Charis L Himeda1, Terrence E O'Brien3, William Thomas4, Baback Roshanravan5, Takako I Jones1, Peter L Jones6
1Department of Pharmacology, Center for Molecular Medicine/MS-0318, University of Nevada, Reno School of Medicine, 1664 N. Virginia St., Reno, NV, 89557, USA.
2The Department of Pathology, Johns Hopkins University School of Medicine, 720 Rutland Ave, Baltimore, MD, 21205, USA.
3Atomwise Inc., San Francisco, CA, 94103, USA.
4, Wyckoff, USA.
5Division of Nephrology, Genome Biomedical Sciences Facility, University of California Davis, 451 Health Sciences Drive, Ste 5321, Sacramento, CA, 95616, USA.
6Department of Pharmacology, Center for Molecular Medicine/MS-0318, University of Nevada, Reno School of Medicine, 1664 N. Virginia St., Reno, NV, 89557, USA. peterjones@med.unr.edu.
Abstract
Facioscapulohumeral muscular dystrophy (FSHD) is caused by epigenetic dysregulation of the disease locus, leading to pathogenic misexpression of DUX4 in skeletal muscle. Thus, most FSHD therapeutic approaches target DUX4. Our previous study identified the chromatin remodeling factor BAZ1A (bromodomain adjacent to zinc finger domain protein 1A) as a promising target for therapeutic development. Her…
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