DMD and Duchenne muscular dystrophy: We previously generated two independent sources of human MPCs from two precisely CRISPR-corrected DMD patient-derived PSC lines (CORR-R3381X and CORR-K2957fs) and demonstrated the restoration of full-length dystrophin (Dp427) in vitro.33,34 In this study, we assess the in vivo regenerative potential of human CORR-R3381X or CORR-K2957fs MPCs compared with skeletal muscle-derived hCD133+ cells11 by encapsulating them in fibrin/Matrigel hydrogels, followed by transplantation into dystrophin-deficient mdx nude mice.