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, M. Nobles, D. Rybin, R. Moccia, A. Kowala, J. Meng, S. Garren, P. Liu, J. E. Morgan, F. Muntoni, N. Christoforou, J. Owens, A. Tinker, 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.
bioRxiv · Feb 17, 2022Cell Biology