Characterization of hiPSC-Derived Muscle Progenitors Reveals Distinctive Markers for Myogenic Cell Purification Toward Cell Therapy

Stem Cell Reports. 2021 Apr 13;16(4):883-898. doi: 10.1016/j.stemcr.2021.03.004. Epub 2021 Apr 1.


The transplantation of muscle progenitor cells (MuPCs) differentiated from human induced pluripotent stem cells (hiPSCs) is a promising approach for treating skeletal muscle diseases such as Duchenne muscular dystrophy (DMD). However, proper purification of the MuPCs before transplantation is essential for clinical application. Here, by using MYF5 hiPSC reporter lines, we identified two markers for myogenic cell purification: CDH13, which purified most of the myogenic cells, and FGFR4, which purified a subset of MuPCs. Cells purified with each of the markers showed high efficiency for regeneration after transplantation and contributed to the restoration of dystrophin expression in DMD-immunodeficient model mice. Moreover, we found that MYF5 regulates CDH13 expression by binding to the promoter regions. These findings suggest that FGFR4 and CDH13 are strong candidates for the purification of hiPSC-derived MuPCs for therapeutical application.

Keywords: CDH13; FGFR4; iPSC; muscle stem cell; skeletal muscle; surface marker.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Base Sequence
  • Biomarkers / metabolism*
  • Cadherins / genetics
  • Cadherins / metabolism
  • Cell Line
  • Cell Separation*
  • Cell- and Tissue-Based Therapy*
  • Gene Expression Regulation
  • Genes, Reporter
  • Induced Pluripotent Stem Cells / cytology*
  • Mice, Transgenic
  • Muscle Development*
  • Muscle, Skeletal / cytology*
  • Myogenic Regulatory Factor 5
  • PAX7 Transcription Factor / metabolism
  • RNA-Seq
  • Receptor, Fibroblast Growth Factor, Type 4 / metabolism
  • Regeneration
  • Stem Cells / cytology*
  • Transcription, Genetic
  • Transcriptome / genetics


  • Biomarkers
  • Cadherins
  • H-cadherin
  • Myf5 protein, mouse
  • Myogenic Regulatory Factor 5
  • PAX7 Transcription Factor
  • Fgfr4 protein, mouse
  • Receptor, Fibroblast Growth Factor, Type 4