Cell BiologybioRxiv
CRISPR-mediated correction of skeletal muscle Ca2+ handling in a novel DMD patient-derived pluripotent stem cell model
C. Morera, J. Kim, A. Paredes-Redondo…·Y.-Y. Lin
This study uses CRISPR to correct dystrophin mutations in DMD patient-derived PSCs, revealing impaired Ca2+ handling and showing rescue through gene correction.