{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Azzag K"],"funding":["NIAID NIH HHS","National Institutes of Health","NIAMS NIH HHS","Duchenne Research Fund","NIH HHS"],"pagination":["5099-5117"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12338975"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["33(10)"],"pubmed_abstract":["Pluripotent stem cell (PSC)-derived therapies are in clinical trials of terminally differentiated or transiently required cell types, but to date no PSC-derived trial contributing tissue-specific stem cells or any PSC-based skeletal muscle regeneration trial has been approved. We describe a process in accordance with the Current Good Manufacturing Practice (CGMP) to generate large-scale cryopreserved PAX7-induced myogenic progenitors, which reconstitute both fibers and satellite cells, from PSCs. We subjected the clinical-grade cell product MyoPAXon to biodistribution, toxicity, and tumorigenicity studies in mice under Good Laboratory Practice conditions with no adverse effects and demonstrate long-term engraftment (>1 year) and efficacy in dystrophic mice. Transplantation of 37-60 million"],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pubmed_title":["Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies."],"pmcid":["PMC12338975"],"funding_grant_id":["R34 AR081536","R01 AR081882","R01 AR078571","U24 AI126683","P40 OD028116","R01 AR078624"],"pubmed_authors":["Norris B","Flory C","Ramachandran S","Kim H","Seelig D","Kyba M","Kiley J","Perlingeiro RCR","Hocum Stone LL","Everett J","Shappa Faustich J","Wagner JE","Ahlquist A","Schumacher RJ","Magli A","Kang PB","Rangarajan P","Bushman F","McKenna DH","Azzag K","Sumstad D","Kadidlo D","Crist SB","Graham ML"],"additional_accession":[]},"is_claimable":false,"name":"Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies.","description":"Pluripotent stem cell (PSC)-derived therapies are in clinical trials of terminally differentiated or transiently required cell types, but to date no PSC-derived trial contributing tissue-specific stem cells or any PSC-based skeletal muscle regeneration trial has been approved. We describe a process in accordance with the Current Good Manufacturing Practice (CGMP) to generate large-scale cryopreserved PAX7-induced myogenic progenitors, which reconstitute both fibers and satellite cells, from PSCs. We subjected the clinical-grade cell product MyoPAXon to biodistribution, toxicity, and tumorigenicity studies in mice under Good Laboratory Practice conditions with no adverse effects and demonstrate long-term engraftment (>1 year) and efficacy in dystrophic mice. Transplantation of 37-60 million","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Oct","modification":"2026-07-15T13:13:30.338Z","creation":"2026-07-05T03:08:14.213Z"},"accession":"S-EPMC12338975","cross_references":{"pubmed":["40682272"],"doi":["10.1016/j.ymthe.2025.07.007"]}}