Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors

Jaemin Kim, Alessandro Magli, Sunny S.K. Chan, Vanessa K.P. Oliveira, Jianbo Wu, Radbod Darabi, Michael Kyba, Rita C.R. Perlingeiro

Research output: Contribution to journalArticlepeer-review

47 Scopus citations

Abstract

Recent reports have documented the differentiation of human pluripotent stem cells toward the skeletal myogenic lineage using transgene- and cell purification-free approaches. Although these protocols generate myocytes, they have not demonstrated scalability, safety, and in vivo engraftment, which are key aspects for their future clinical application. Here we recapitulate one prominent protocol, and show that it gives rise to a heterogeneous cell population containing myocytes and other cell types. Upon transplantation, the majority of human donor cells could not contribute to myofiber formation. As a proof-of-principle, we incorporated the inducible PAX7 lentiviral system into this protocol, which then enabled scalable expansion of a homogeneous population of skeletal myogenic progenitors capable of forming myofibers in vivo. Our findings demonstrate the methods for scalable expansion of PAX7+ myogenic progenitors and their purification are critical for practical application to cell replacement treatment of muscle degenerative diseases.

Original languageEnglish (US)
Pages (from-to)12-22
Number of pages11
JournalStem Cell Reports
Volume9
Issue number1
DOIs
StatePublished - Jul 11 2017

Bibliographical note

Publisher Copyright:
© 2017 The Author(s)

Keywords

  • MHC myocytes
  • PAX7 myogenic progenitors
  • engraftment
  • morphogens
  • pluripotent stem cells
  • purification
  • skeletal muscle
  • stem cell therapy
  • transgene

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