Restoration of muscle fibers and satellite cells after isogenic MSC transplantation with microdystrophin gene delivery

► mMSCs with microdystrophy delivery were transduced efficiently by retrovirus. ► Infusion of mMSCs with gene direction could partly restoration myofibers of mdx mice. ► Implanted mMSCs were capable of long-term survival as muscle satellite cells. Duchenne muscular dystrophy is the most prevalent inheritable muscle disease. Transplantation of autologous stem cells with gene direction is an ideal therapeutic approach for the disease. The current study aimed to investigate the restoration of myofibers in mdx mice after mdx bone marrow-derived mesenchymal stem cell (mMSC) transplantation with human microdystrophin delivery. Possible mechanisms of action were also studied. In our research, mMSCs were successfully transduced by retrovirus carrying a functional human microdystrophin gene. Transplantation of transduced mMSCs enabled persistent dystrophin restoration in the skeletal muscle of mdx mice up to the 12th week after transplantation. Simultaneous coexpression of human microdystrophin and desmin showed that implanted mMSCs are capable of long-term survival as muscle satellite cells.