Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic Progenitors

Skeletal muscle regeneration requires a reliable source of myogenic progenitor cells capable of forming new fibers and creating a self-renewing satellite cell pool. Human induced pluripotent stem cell (hiPSC)-derived teratomas have emerged as a novel in vivo platform for generating skeletal myogenic...

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Main Authors: Zahra Khosrowpour, Nivedha Ramaswamy, Elise N. Engquist, Berkay Dincer, Alisha M. Shah, Hossam A. N. Soliman, Natalya A. Goloviznina, Peter I. Karachunski, Michael Kyba
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Language:English
Published: MDPI AG 2025-07-01
Series:Cells
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Online Access:https://www.mdpi.com/2073-4409/14/15/1150
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author Zahra Khosrowpour
Nivedha Ramaswamy
Elise N. Engquist
Berkay Dincer
Alisha M. Shah
Hossam A. N. Soliman
Natalya A. Goloviznina
Peter I. Karachunski
Michael Kyba
author_facet Zahra Khosrowpour
Nivedha Ramaswamy
Elise N. Engquist
Berkay Dincer
Alisha M. Shah
Hossam A. N. Soliman
Natalya A. Goloviznina
Peter I. Karachunski
Michael Kyba
author_sort Zahra Khosrowpour
collection DOAJ
description Skeletal muscle regeneration requires a reliable source of myogenic progenitor cells capable of forming new fibers and creating a self-renewing satellite cell pool. Human induced pluripotent stem cell (hiPSC)-derived teratomas have emerged as a novel in vivo platform for generating skeletal myogenic progenitors, although in vivo studies to date have provided only an early single-time-point snapshot. In this study, we isolated a specific population of CD82<sup>+</sup> ERBB3<sup>+</sup> NGFR<sup>+</sup> cells from human iPSC-derived teratomas and verified their long-term in vivo regenerative capacity following transplantation into NSG-mdx<sup>4Cv</sup> mice. Transplanted cells engrafted, expanded, and generated human Dystrophin<sup>+</sup> muscle fibers that increased in size over time and persisted stably long-term. A dynamic population of PAX7<sup>+</sup> human satellite cells was established, initially expanding post-transplantation and declining moderately between 4 and 8 months as fibers matured. MyHC isoform analysis revealed a time-based shift from embryonic to neonatal and slow fiber types, indicating a slow progressive maturation of the graft. We further show that these progenitors can be cryopreserved and maintain their engraftment potential. Together, these findings give insight into the evolution of teratoma-derived human myogenic stem cell grafts, and highlight the long-term regenerative potential of teratoma-derived human skeletal myogenic progenitors.
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spelling doaj-art-d0746e62748745a385f4163507ef2f2b2025-08-20T03:02:55ZengMDPI AGCells2073-44092025-07-011415115010.3390/cells14151150Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic ProgenitorsZahra Khosrowpour0Nivedha Ramaswamy1Elise N. Engquist2Berkay Dincer3Alisha M. Shah4Hossam A. N. Soliman5Natalya A. Goloviznina6Peter I. Karachunski7Michael Kyba8Lillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USALillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USALillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USALillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USALillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USALillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USALillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USAGreg Marzolf Jr. Muscular Dystrophy Center, University of Minnesota, Minneapolis, MN 55455, USALillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USASkeletal muscle regeneration requires a reliable source of myogenic progenitor cells capable of forming new fibers and creating a self-renewing satellite cell pool. Human induced pluripotent stem cell (hiPSC)-derived teratomas have emerged as a novel in vivo platform for generating skeletal myogenic progenitors, although in vivo studies to date have provided only an early single-time-point snapshot. In this study, we isolated a specific population of CD82<sup>+</sup> ERBB3<sup>+</sup> NGFR<sup>+</sup> cells from human iPSC-derived teratomas and verified their long-term in vivo regenerative capacity following transplantation into NSG-mdx<sup>4Cv</sup> mice. Transplanted cells engrafted, expanded, and generated human Dystrophin<sup>+</sup> muscle fibers that increased in size over time and persisted stably long-term. A dynamic population of PAX7<sup>+</sup> human satellite cells was established, initially expanding post-transplantation and declining moderately between 4 and 8 months as fibers matured. MyHC isoform analysis revealed a time-based shift from embryonic to neonatal and slow fiber types, indicating a slow progressive maturation of the graft. We further show that these progenitors can be cryopreserved and maintain their engraftment potential. Together, these findings give insight into the evolution of teratoma-derived human myogenic stem cell grafts, and highlight the long-term regenerative potential of teratoma-derived human skeletal myogenic progenitors.https://www.mdpi.com/2073-4409/14/15/1150skeletal muscleregenerationteratomatransplantationxenograftsatellite cells
spellingShingle Zahra Khosrowpour
Nivedha Ramaswamy
Elise N. Engquist
Berkay Dincer
Alisha M. Shah
Hossam A. N. Soliman
Natalya A. Goloviznina
Peter I. Karachunski
Michael Kyba
Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic Progenitors
Cells
skeletal muscle
regeneration
teratoma
transplantation
xenograft
satellite cells
title Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic Progenitors
title_full Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic Progenitors
title_fullStr Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic Progenitors
title_full_unstemmed Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic Progenitors
title_short Long-Term Engraftment and Satellite Cell Expansion from Human PSC Teratoma-Derived Myogenic Progenitors
title_sort long term engraftment and satellite cell expansion from human psc teratoma derived myogenic progenitors
topic skeletal muscle
regeneration
teratoma
transplantation
xenograft
satellite cells
url https://www.mdpi.com/2073-4409/14/15/1150
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