Functional myogenic engraftment from mouse iPS cells

Stem Cell Rev Rep. 2011 Nov;7(4):948-57. doi: 10.1007/s12015-011-9258-2.


Direct reprogramming of adult fibroblasts to a pluripotent state has opened new possibilities for the generation of patient- and disease-specific stem cells. However the ability of induced pluripotent stem (iPS) cells to generate tissue that mediates functional repair has been demonstrated in very few animal models of disease to date. Here we present the proof of principle that iPS cells may be used effectively for the treatment of muscle disorders. We combine the generation of iPS cells with conditional expression of Pax7, a robust approach to derive myogenic progenitors. Transplantation of Pax7-induced iPS-derived myogenic progenitors into dystrophic mice results in extensive engraftment, which is accompanied by improved contractility of treated muscles. These findings demonstrate the myogenic regenerative potential of iPS cells and provide rationale for their future therapeutic application for muscular dystrophies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cardiotoxins / adverse effects
  • Cell Differentiation
  • Cells, Cultured
  • Disease Models, Animal
  • Doxycycline / pharmacology
  • Dystrophin / metabolism
  • Induced Pluripotent Stem Cells / cytology*
  • Induced Pluripotent Stem Cells / drug effects
  • Induced Pluripotent Stem Cells / metabolism
  • Induced Pluripotent Stem Cells / transplantation
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred mdx
  • Muscle Contraction
  • Muscle, Skeletal / cytology
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / transplantation*
  • Muscular Dystrophy, Duchenne / metabolism
  • Muscular Dystrophy, Duchenne / therapy
  • PAX7 Transcription Factor / genetics
  • PAX7 Transcription Factor / metabolism*
  • Regeneration
  • Stem Cell Transplantation
  • Teratoma / metabolism
  • Teratoma / pathology
  • Transgenes


  • Cardiotoxins
  • Dystrophin
  • PAX7 Transcription Factor
  • Pax7 protein, mouse
  • Doxycycline