Cell therapy for duchenne muscular dystrophy using induced pluripotent stem cell-derived muscle stem cells and the potential of regenerative rehabilitation

Abstract

Duchenne muscular dystrophy (DMD) is a hereditary disease characterized by severe muscle weakness resulting from DYSTROPHIN deficiency-associated damage. Muscle regeneration therapy using skeletal muscle stem cell (MuSC) transplantation has demonstrated great promise. Optimized differentiation methods yield efficient induced pluripotent stem cell-derived MuSCs (iMuSCs); transplanted iMuSCs have high regenerative potential in DMD mouse models. However, achieving complete pathophysiological recovery of DMD motor function through cell therapy alone is challenging. According to recent studies, exercise can significantly improve DMD pathophysiology. Current research aims to enhance therapeutic efficacy by combining cell transplantation with exercise interventions, addressing the limitations of cell-transplantation therapy for DMD.