Identification of compounds that repress DUX4 expression in facioscapulohumeral muscular dystrophy

Abstract excerpt

<h4>ABSTRACT</h4> 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...

Topics

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• Combined Modality Therapy
• Disease Management
• Disease Susceptibility
• Drug Development
• Drug Evaluation, Preclinical
• Molecular Targeted Therapy
• Muscle Physiology and Disorders
• Muscular Dystrophy, Facioscapulohumeral

Identifiers and source

Literature Corpus work

cb66c067-3558-5cc8-9680-4e3778aaa5d7

DOI

10.64898/2026.03.09.710626