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Double homeobox 4 (DUX4) is a transcription factor that regulates gene expression during early embryonic development. In healthy tissue, DUX4 is normally silenced but is aberrantly expressed in facioscapulohumeral muscular dystrophy (FSHD), leading to muscle wasting. Due to its role in transcriptional regulation and disease, DUX4 is considered a therapeutic target. Potential therapeutic interventions involve modulating its expression or inhibiting its activity.
Modulation of DUX4 expression or activity
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