Transplantation of hPSC-derived skeletal muscle progenitor cells for muscular dystrophy therapies

The functional and therapeutic potential of human fetal, adult, and hPSC derived skeletal muscle progenitor cells (SMPCs) in vivo is not known. We are identifying the most engraftable cell type, signaling factors, and niche components to increase the potential of cell based therapy using SMPCs. This work will be combined with CRIPSR/Cas9 gene editing to develop personalized approaches for patients with muscle disease.