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Development of cell therapy for muscular dystrophy by iPSC-derived muscle stem cell

Cell therapy is one of desired method for treating intractable muscular diseases, such as Duchenne muscular dystrophy (DMD). Here, we demonstrated the effective stepwise differentiation method from human iPSCs to engraftable muscle stem cells without transgene induction. We induced myotome-like population that is identified as Myf5 positive cells, which showed highly myogenic differentiation potential in vitro. Gene expression profile of purified Myf5+ cells demonstrated that the expression of Pax7 was significantly increased in Myf5+ cells at the late stage of differentiation. To assess the regeneration potential, we transplanted the Myf5+ cells at the late stage of differentiation into immunodeficient DMD-model mice. The Myf5+ cells could be engrafted in more than one hundred of host myofibers and regenerate the diseased muscles with producing dystrophin. Finally, we confirmed the recovery of muscle function after transplantation. Taken together, we demonstrate that the transplantation of the human iPSC-derived muscle stem cells with step-wise differentiation can be effective for DMD with amelioration of muscle function.