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Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies
Although skeletal muscle cells can be generated from human induced pluripotent stem cells (iPSCs), transgene-free protocols include only limited options for their purification and expansion. In this study, we found that fluorescence-activated cell sorting-purified myogenic progenitors generated from...
Autores principales: | , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5993675/ https://www.ncbi.nlm.nih.gov/pubmed/29731431 http://dx.doi.org/10.1016/j.stemcr.2018.04.002 |
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author | van der Wal, Erik Herrero-Hernandez, Pablo Wan, Raymond Broeders, Mike in 't Groen, Stijn L.M. van Gestel, Tom J.M. van IJcken, Wilfred F.J. Cheung, Tom H. van der Ploeg, Ans T. Schaaf, Gerben J. Pijnappel, W.W.M. Pim |
author_facet | van der Wal, Erik Herrero-Hernandez, Pablo Wan, Raymond Broeders, Mike in 't Groen, Stijn L.M. van Gestel, Tom J.M. van IJcken, Wilfred F.J. Cheung, Tom H. van der Ploeg, Ans T. Schaaf, Gerben J. Pijnappel, W.W.M. Pim |
author_sort | van der Wal, Erik |
collection | PubMed |
description | Although skeletal muscle cells can be generated from human induced pluripotent stem cells (iPSCs), transgene-free protocols include only limited options for their purification and expansion. In this study, we found that fluorescence-activated cell sorting-purified myogenic progenitors generated from healthy controls and Pompe disease iPSCs can be robustly expanded as much as 5 × 10(11)-fold. At all steps during expansion, cells could be cryopreserved or differentiated into myotubes with a high fusion index. In vitro, cells were amenable to maturation into striated and contractile myofibers. Insertion of acid α-glucosidase cDNA into the AAVS1 locus in iPSCs using CRISPR/Cas9 prevented glycogen accumulation in myotubes generated from a patient with classic infantile Pompe disease. In vivo, the expression of human-specific nuclear and sarcolemmar antigens indicated that myogenic progenitors engraft into murine muscle to form human myofibers. This protocol is useful for modeling of skeletal muscle disorders and for using patient-derived, gene-corrected cells to develop cell-based strategies. |
format | Online Article Text |
id | pubmed-5993675 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | Elsevier |
record_format | MEDLINE/PubMed |
spelling | pubmed-59936752018-06-11 Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies van der Wal, Erik Herrero-Hernandez, Pablo Wan, Raymond Broeders, Mike in 't Groen, Stijn L.M. van Gestel, Tom J.M. van IJcken, Wilfred F.J. Cheung, Tom H. van der Ploeg, Ans T. Schaaf, Gerben J. Pijnappel, W.W.M. Pim Stem Cell Reports Resource Although skeletal muscle cells can be generated from human induced pluripotent stem cells (iPSCs), transgene-free protocols include only limited options for their purification and expansion. In this study, we found that fluorescence-activated cell sorting-purified myogenic progenitors generated from healthy controls and Pompe disease iPSCs can be robustly expanded as much as 5 × 10(11)-fold. At all steps during expansion, cells could be cryopreserved or differentiated into myotubes with a high fusion index. In vitro, cells were amenable to maturation into striated and contractile myofibers. Insertion of acid α-glucosidase cDNA into the AAVS1 locus in iPSCs using CRISPR/Cas9 prevented glycogen accumulation in myotubes generated from a patient with classic infantile Pompe disease. In vivo, the expression of human-specific nuclear and sarcolemmar antigens indicated that myogenic progenitors engraft into murine muscle to form human myofibers. This protocol is useful for modeling of skeletal muscle disorders and for using patient-derived, gene-corrected cells to develop cell-based strategies. Elsevier 2018-05-03 /pmc/articles/PMC5993675/ /pubmed/29731431 http://dx.doi.org/10.1016/j.stemcr.2018.04.002 Text en © 2018 The Author(s) http://creativecommons.org/licenses/by/4.0/ This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Resource van der Wal, Erik Herrero-Hernandez, Pablo Wan, Raymond Broeders, Mike in 't Groen, Stijn L.M. van Gestel, Tom J.M. van IJcken, Wilfred F.J. Cheung, Tom H. van der Ploeg, Ans T. Schaaf, Gerben J. Pijnappel, W.W.M. Pim Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies |
title | Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies |
title_full | Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies |
title_fullStr | Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies |
title_full_unstemmed | Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies |
title_short | Large-Scale Expansion of Human iPSC-Derived Skeletal Muscle Cells for Disease Modeling and Cell-Based Therapeutic Strategies |
title_sort | large-scale expansion of human ipsc-derived skeletal muscle cells for disease modeling and cell-based therapeutic strategies |
topic | Resource |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5993675/ https://www.ncbi.nlm.nih.gov/pubmed/29731431 http://dx.doi.org/10.1016/j.stemcr.2018.04.002 |
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