Journal
STEM CELL REPORTS
Volume 16, Issue 4, Pages 883-898Publisher
CELL PRESS
DOI: 10.1016/j.stemcr.2021.03.004
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Funding
- Core Center for iPS Cell Research by the Japan Agency for Medical Research and Development, AMED [19bm0104001h0007]
- Acceleration Program for Intractable Diseases Research Utilizing Disease-Specific iPS Cells by the Japan Agency for Medical Research and Development, AMED [19bm0804005h0003]
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CDH13 and FGFR4 are identified as markers for purification of hiPSC-derived MuPCs, which show high efficiency for regeneration after transplantation and contribute to the restoration of dystrophin expression in DMD-immunodeficient model mice.
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.
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