Targeted DUX4 base editing improves muscle function in an iPSC-derived model of childhood-onset FSHD
Peter J. Houweling, Vanessa G. Crossman, Leonit Kiriaev, Kathrin Mattes, Chrystal F. Tiong, Chantal A. Coles, Claire Hogan, Natasha Tuano, Richard J. Mills, Sara E. Howden, Katy de Valle, Kathryn N. North, Ian R. Woodcock
This study uses iPSC-derived muscle models to show that DUX4 base editing improves muscle function in childhood-onset FSHD.
bioRxiv · Jul 29, 2026 · PreprintCell Biology