Exosome-Derived Dystrophin from Allograft Myogenic Progenitors Improves Cardiac Function in Duchenne Muscular Dystrophic Mice

Progressive cardiomyocyte loss in Duchenne muscular dystrophy (DMD) leads to cardiac fibrosis, cardiomyopathy, and eventually heart failure. In the present study,we observed thatmyogenic progenitor cells (MPC) carrymRNAfor the dystrophin gene.We tested whether cardiac function can be improved inDMDby allograft transplantation of MPC-derived exosomes (MPC-Exo) into the heart to restore dystrophin protein expression. Exo fromC2C12 cells (anMPC cell line) or vehiclewere delivered locally into the hearts of MDXmice. After 2 days of treatment,we observed thatMPC-Exo restored dystrophin expression in the hearts ofMDXmice, which correlated with improved myocardial function in dystrophin-deficient MDX mouse hearts. In conclusion, this study demonstrated that allogeneic WT-MPC-Exo transplantation transiently restored dystrophin gene expression and improved cardiac function in MDX mice, suggesting that allogenic exosomal delivery may serve as an alternative treatment for cardiomyopathy of DMD.