News Releases & Research Results Effectiveness of transplantation of skeletal muscle stem cells derived from human induced pluripotent stem cells in muscular dystrophy mouse model

The results of collaborative research conducted by Researcher Mingming Zhao and Associate Professor Hidetoshi Sakurai of the Department of Clinical Application, Center for iPS Cell Research and Application (CiRA), Kyoto University; and others.

• Duchenne muscular dystrophy (DMD) is a serious progressive muscular disease caused by the deficiency of a muscular protein called DYSTROPHIN. Cell transplantation therapy has been proposed as a method to regenerate DYSTROPHIN in the skeletal muscle.
• This time, the research group succeeded in the differentiation of skeletal muscle stem cells, which have a high potential to regenerate the muscle tissue, from human induced pluripotent stem (iPS) cells.
• Furthermore, the transplantation of the skeletal muscle stem cells differentiated from human iPS cells to DMD model mice improved their muscle tension.
• The results of this research project form the basis for the establishment of a method for stable production of cells with high therapeutic efficacy.

This research project was conducted with the support of the Research Center Network for Realization of Regenerative Medicine by AMED.

The results of research were published online in Stem Cell Reports, an American scientific journal, on July 3.

Mingming Zhao., et al. Induced Fetal Human Muscle Stem Cells with High Therapeutic Potential in a Mouse Muscular Dystrophy Model Stem Cell Reports
DOI：10.1016/j.stemcr.2020.06.004