Initial failure in myoblast transplantation therapy has led the way toward the isolation of muscle stem cells: potential for tissue regeneration.

Myoblast transfer therapy can restore dystrophin expressing myofibers in mdx mice and patients with Duchenne muscular dystrophy (DMD). However, the effectiveness of this technique is hindered by numerous limitations, including minimal distribution of cells after injection, immune rejection, and poor cell survival. Initial studies revealed that only a small population of cells was responsible for muscle regeneration. Compared with myoblast transplantation, the injection of a population of myogenic cells purified with the pre-plate technique results in a superior regeneration of dystrophin-expressing myofibers. These postnatal muscle-derived stem cells (MDSC) undergo self-renewal, display long-term proliferation, and differentiate into multiple lineages. This review examines the initial obstacles encountered in myoblast transplantation, the regenerative properties of MDSC, and the potential use of these stem cells not only for DMD therapy but also for multiple applications, including bone repair and blood reconstitution.