Derivation of {Skeletal} {Myogenic} {Precursors} from {Human} {Pluripotent} {Stem} {Cells} {Using} {Conditional} {Expression} of {PAX}7 - {Springer}

Abstract

Cell-based therapies are considered as one of the most promising approachesfor the treatment of degenerating pathologies including muscle disordersand dystrophies. Advances in the approach of reprogramming somaticcells into induced pluripotent stem (iPS) cells allow for the possibilityof using the patient’s own pluripotent cells to generate specifictissues for autologous transplantation. In addition, patient-specifictissue derivatives have been shown to represent valuable materialfor disease modeling and drug discovery. Nevertheless, directed differentiationof pluripotent stem cells into a specific lineage is not a trivialtask especially in the case of skeletal myogenesis, which is generallypoorly recapitulated during the in vitro differentiation of pluripotentstem cells. Here, we describe a practical and efficient method forthe derivation of skeletal myogenic precursors from differentiatinghuman pluripotent stem cells using controlled expression of PAX7.Flow cytometry (FACS) purified myogenic precursors can be expandedexponentially and differentiated in vitro into myotubes, enablingresearchers to use these cells for disease modeling as well as therapeuticpurposes.