Derivation of Engraftable Myogenic Precursors from Murine ES/iPS cells and Generation of Disease-specific iPS cells from Patients with Duchenne Muscular dystrophy (DMD) and Other Diseases

TL;DRAbstract

Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene, is a progressive, lethal muscle disorder with no effective cure despite extensive research efforts in the field. In recent years, many different myogenic cells originating from adult tissues have been reported. However, the establishment of a reliable cell source is required for clinical application. Embryonic stem (ES) cells and the recently established induced pluripotent stem (iPS) cells are totipotent stem cells that are infinitely expandable and capable of differentiating into all types of somatic cells. In this study, we established a novel protocol to derive myogenic precursors from murine ES iPS cells with a monoclonal antibody SM C-2.6 that recognizes quiescent satellite cells. SM C-2.6-positive cells are highly myogenic and efficiently differentiate into myofibers both in vitro and in vivo. Furthermore, the transplanted cells demonstrated extensive muscle regeneration activity in a second

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