Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors
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 a...
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| Format: | Article |
| Language: | English |
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Elsevier
2017-07-01
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| Series: | Stem Cell Reports |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2213671117301765 |
| _version_ | 1811322640151871488 |
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| author | Jaemin Kim Alessandro Magli Sunny S.K. Chan Vanessa K.P. Oliveira Jianbo Wu Radbod Darabi Michael Kyba Rita C.R. Perlingeiro |
| author_facet | Jaemin Kim Alessandro Magli Sunny S.K. Chan Vanessa K.P. Oliveira Jianbo Wu Radbod Darabi Michael Kyba Rita C.R. Perlingeiro |
| author_sort | Jaemin Kim |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-04-13T13:39:42Z |
| format | Article |
| id | doaj.art-ed612bed99ae47dcac2b1087e212cf7a |
| institution | Directory Open Access Journal |
| issn | 2213-6711 |
| language | English |
| last_indexed | 2024-04-13T13:39:42Z |
| publishDate | 2017-07-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Stem Cell Reports |
| spelling | doaj.art-ed612bed99ae47dcac2b1087e212cf7a2022-12-22T02:44:41ZengElsevierStem Cell Reports2213-67112017-07-0191122210.1016/j.stemcr.2017.04.022Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic ProgenitorsJaemin Kim0Alessandro Magli1Sunny S.K. Chan2Vanessa K.P. Oliveira3Jianbo Wu4Radbod Darabi5Michael Kyba6Rita C.R. Perlingeiro7Department of Medicine, Lillehei Heart Institute, University of Minnesota, 4-128 CCRB, 2231 6th Street South East, Minneapolis, MN 55455, USADepartment of Medicine, Lillehei Heart Institute, University of Minnesota, 4-128 CCRB, 2231 6th Street South East, Minneapolis, MN 55455, USADepartment of Pediatrics, Lillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USADepartment of Medicine, Lillehei Heart Institute, University of Minnesota, 4-128 CCRB, 2231 6th Street South East, Minneapolis, MN 55455, USACenter for Stem Cell and Regenerative Medicine, The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases, University of Texas Health Science Center at Houston, Houston, TX 77030, USACenter for Stem Cell and Regenerative Medicine, The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases, University of Texas Health Science Center at Houston, Houston, TX 77030, USADepartment of Pediatrics, Lillehei Heart Institute, University of Minnesota, Minneapolis, MN 55455, USADepartment of Medicine, Lillehei Heart Institute, University of Minnesota, 4-128 CCRB, 2231 6th Street South East, Minneapolis, MN 55455, USARecent 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.http://www.sciencedirect.com/science/article/pii/S2213671117301765pluripotent stem cellsskeletal musclemorphogenspurificationPAX7+ myogenic progenitorsMHC+ myocytestransgeneengraftmentstem cell therapy |
| spellingShingle | Jaemin Kim Alessandro Magli Sunny S.K. Chan Vanessa K.P. Oliveira Jianbo Wu Radbod Darabi Michael Kyba Rita C.R. Perlingeiro Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors Stem Cell Reports pluripotent stem cells skeletal muscle morphogens purification PAX7+ myogenic progenitors MHC+ myocytes transgene engraftment stem cell therapy |
| title | Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors |
| title_full | Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors |
| title_fullStr | Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors |
| title_full_unstemmed | Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors |
| title_short | Expansion and Purification Are Critical for the Therapeutic Application of Pluripotent Stem Cell-Derived Myogenic Progenitors |
| title_sort | expansion and purification are critical for the therapeutic application of pluripotent stem cell derived myogenic progenitors |
| topic | pluripotent stem cells skeletal muscle morphogens purification PAX7+ myogenic progenitors MHC+ myocytes transgene engraftment stem cell therapy |
| url | http://www.sciencedirect.com/science/article/pii/S2213671117301765 |
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