骨格筋再生における細胞移植とリハビリテーション

抄録

Duchenne muscular dystrophy (DMD) is one of the most severe forms of muscle disorders. Muscle in DMD patients is extremely fragile and can be damaged even during normal daily activity. The disease is caused by mutations in the DMD gene that result in the loss of dystrophin protein expression. There is little in the way of treatment for the disease and no cure. Some investigators have been developing cell therapies for DMD by generating muscle stem cells from human induced pluripotent stem (iPS) cells and other progenitor/stem cells. Although reports have shown the effects of cell transplantation therapy in DMD, optimal methods that maximize the efficacy of the transplantation are still needed. Exercise is expected to enhance the effect of cell therapy for DMD, acting as a form of “regenerative rehabilitation”. We evaluated the efficacy of transplanting human myogenic progenitor cells that can express dystrophin into the skeletal muscle of DMD model mice by histological and functional analysis. We found that optimized muscle contraction training programs enhance the effect of the cell transplantation therapy. Regenerative rehabilitation could be a basis for effective cell therapy towards DMD patients.