Health AI Central
Share:
Peer-Reviewed Publication
bioRxiv2026March 17, 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 Thomas1,2,3,4, Baback Roshanravan4, Takako I Jones1, Peter L Jones1
1The Department of Pharmacology, University of Nevada, Reno School of Medicine, 1664 N. Virginia St, Reno, NV 89557, USA.
2Current Address: The Department of Pathology, Johns Hopkins University School of Medicine, 720 Rutland Ave, Baltimore, MD 21205, USA.
3Atomwise Inc., San Francisco, CA 94103, USA.
4Division of Nephrology, University of California Davis, Genome Biomedical Sciences Facility, 451 Health Sciences Drive, Ste 5321, Sacramento, CA 95616, USA.
PubMed PMC DOI PDF

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…

Create a free account to keep reading

Free members get 10 full research views every month across publications, clinical trials, FDA clearances, adverse events, and NIH grants. No credit card required.

Create Free AccountSign In

Want unlimited research access? See Pro plans

Data Accuracy Notice: Research intelligence on Health AI Central is aggregated from public sources (PubMed, ClinicalTrials.gov, FDA, NIH, CMS, and others) and refreshed nightly. Classifications and derived metrics are produced by automated methods described in our Methodology. We recommend verifying critical data points against the primary sources before making decisions.