Embryonic/pluripotent stem cell-derived myogenic progenitor cells for Duchenne muscular dystrophy

The aim of this study is to successfully derive large quantities of myogenic precursor cells, for treatment of Duchenne muscular dystrophy, by conditionally expressing PAX7 in human ES/iPS cells. Human embryonic stem cells (ES) or human induced pluripotent stem cells (iPS) were initially differentiated by PAX7 into myogenic progenitor cells. These progenitors were then transplanted into NSG-mdx mice. This mice model lacks dystrophin and B,T, and NK cells. The transplantation led to recovery of muscle function.