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, Katherine Xu, Vanessa Chiprez Meza, Ayushi Mohanty, María Fernanda Velásquez, Michael Liu, Alex Engel, 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.
bioRxiv · Aug 20, 2026 · PreprintBioengineering