BioengineeringbioRxivPreprint
In vitro and in silico characterization of competitive inhibition and repression of DUX4 target gene activation as a therapeutic approach for facioscapulohumeral muscular dystrophy (FSHD)
Heloise M. Hoffmann, Alice Finkelstein, Amanuel Worku Geremew…·Phillip Kyriakakis
This paper demonstrates that a DUX4 DNA-binding domain fused to a human KRAB domain can effectively repress DUX4-FL transcriptional activity in vitro, and develops computational models to explore its therapeutic potential for FSHD.
DOI 10.64898/2026.08.04.742607