Two articles from two independent groups, Japanese and Korean, illustrate the difficulties faced in preclinical studies of cell therapy in Duchenne muscular dystrophy (DMD):
- the Japanese study demonstrates, in both the mouse and rat models of DMD, that intraperitoneal or transplacental transplants of myoblasts or stem cells of mesenchymal origin do not take place, even if they are done in utero
- the Korean study consisting of injecting stem cells of tonsil origin only into mdx mice is a little more encouraging, with a genuine re-expression of dystrophin in the transplanted muscles and a definite functional improvement.
Thus, there is still a long way to go before we see clinical applications of cell therapy in DMD.
