title: Functional myogenic engraftment from mouse iPS cells.
abstract: 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.
authors: Lillehei Heart Institute, Department of Medicine, University of Minnesota, 4-124 Nils Hasselmo Hall, 312 Church St. S.E., Minneapolis, 55455 MN, USA.
authors: Radbod
authors: R
authors: Darabi
authors: Weihong
authors: W
authors: Pan
authors: Darko
authors: D
authors: Bosnakovski
authors: June
authors: J
authors: Baik
authors: Michael
authors: M
authors: Kyba
authors: Rita C R
authors: RC
authors: Perlingeiro
mesh_terms: D000818:Animals; D054715:Cardiotoxins / Q000009:adverse effects; D002454:Cell Differentiation; D002478:Cells, Cultured; D004195:Disease Models, Animal; D004318:Doxycycline / Q000494:pharmacology; D016189:Dystrophin / Q000378:metabolism; D057026:Induced Pluripotent Stem Cells / Q000166:cytology* / Q000187:drug effects / Q000378:metabolism / Q000637:transplantation; D051379:Mice; D008810:Mice, Inbred C57BL; D018101:Mice, Inbred mdx; D009119:Muscle Contraction; D018482:Muscle, Skeletal / Q000166:cytology / Q000378:metabolism / Q000637:transplantation*; D020388:Muscular Dystrophy, Duchenne / Q000378:metabolism / Q000628:therapy; D051763:PAX7 Transcription Factor / Q000235:genetics / Q000378:metabolism*; D012038:Regeneration; D033581:Stem Cell Transplantation; D013724:Teratoma / Q000378:metabolism / Q000473:pathology; D019076:Transgenes
chemical_list: D054715:Cardiotoxins; D016189:Dystrophin; D051763:PAX7 Transcription Factor; C497322:Pax7 protein, mouse; D004318:Doxycycline