Hit-and-run silencing of endogenous DUX4 by targeting DNA hypomethylation on D4Z4 repeats in facioscapulohumeral muscular dystrophy
M. Sasaki-Honda, T. Jonouchi, M. Arai, J. He, K. Okita, S. Sakurai, T. Yamamoto, H. Sakurai
This study uses hit-and-run silencing to suppress DUX4 in FSHD patient-derived iPSCs by targeting DNA hypomethylation on D4Z4 repeats, achieving sustained suppression for at least two weeks.
bioRxiv · Apr 12, 2022 · PreprintPathology