{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,15]],"date-time":"2026-08-15T01:03:39Z","timestamp":1786755819229,"version":"build-2736575974"},"reference-count":219,"publisher":"MDPI AG","issue":"7","license":[{"start":{"date-parts":[[2024,3,29]],"date-time":"2024-03-29T00:00:00Z","timestamp":1711670400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cells"],"abstract":"<jats:p>Cell therapies derived from induced pluripotent stem cells (iPSCs) offer a promising avenue in the field of regenerative medicine due to iPSCs\u2019 expandability, immune compatibility, and pluripotent potential. An increasing number of preclinical and clinical trials have been carried out, exploring the application of iPSC-based therapies for challenging diseases, such as muscular dystrophies. The unique syncytial nature of skeletal muscle allows stem\/progenitor cells to integrate, forming new myonuclei and restoring the expression of genes affected by myopathies. This characteristic makes genome-editing techniques especially attractive in these therapies. With genetic modification and iPSC lineage specification methodologies, immune-compatible healthy iPSC-derived muscle cells can be manufactured to reverse the progression of muscle diseases or facilitate tissue regeneration. Despite this exciting advancement, much of the development of iPSC-based therapies for muscle diseases and tissue regeneration is limited to academic settings, with no successful clinical translation reported. The unknown differentiation process in vivo, potential tumorigenicity, and epigenetic abnormality of transplanted cells are preventing their clinical application. In this review, we give an overview on preclinical development of iPSC-derived myogenic cell transplantation therapies including processes related to iPSC-derived myogenic cells such as differentiation, scaling-up, delivery, and cGMP compliance. And we discuss the potential challenges of each step of clinical translation. Additionally, preclinical model systems for testing myogenic cells intended for clinical applications are described.<\/jats:p>","DOI":"10.3390\/cells13070596","type":"journal-article","created":{"date-parts":[[2024,4,1]],"date-time":"2024-04-01T05:44:36Z","timestamp":1711950276000},"page":"596","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":13,"title":["Challenges and Considerations of Preclinical Development for iPSC-Based Myogenic Cell Therapy"],"prefix":"10.3390","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6821-385X","authenticated-orcid":false,"given":"Congshan","family":"Sun","sequence":"first","affiliation":[{"name":"Vita Therapeutics, Baltimore, MD 21043, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carlo","family":"Serra","sequence":"additional","affiliation":[{"name":"Department of Neurology, Baylor College of Medicine, Houston, TX 77030, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brianna Harley","family":"Kalicharan","sequence":"additional","affiliation":[{"name":"Vita Therapeutics, Baltimore, MD 21043, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Harding","sequence":"additional","affiliation":[{"name":"Vita Therapeutics, Baltimore, MD 21043, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mahendra","family":"Rao","sequence":"additional","affiliation":[{"name":"Vita Therapeutics, Baltimore, MD 21043, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2024,3,29]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1038\/309255a0","article-title":"Formation of germ-line chimaeras from embryo-derived teratocarcinoma cell lines","volume":"309","author":"Bradley","year":"1984","journal-title":"Nature"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1016\/j.cell.2006.07.024","article-title":"Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors","volume":"126","author":"Takahashi","year":"2006","journal-title":"Cell"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1016\/j.cell.2007.11.019","article-title":"Induction of pluripotent stem cells from adult human fibroblasts by defined factors","volume":"131","author":"Takahashi","year":"2007","journal-title":"Cell"},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"1917","DOI":"10.1126\/science.1151526","article-title":"Induced pluripotent stem cell lines derived from human somatic cells","volume":"318","author":"Yu","year":"2007","journal-title":"Science"},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1073\/pnas.0811426106","article-title":"Reprogramming of murine and human somatic cells using a single polycistronic vector","volume":"106","author":"Carey","year":"2009","journal-title":"Proc. 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