{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T05:20:35Z","timestamp":1790832035451,"version":"4.1.0"},"reference-count":37,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2023,4,10]],"date-time":"2023-04-10T00:00:00Z","timestamp":1681084800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["JP18im0210817 JP13bm0104001"],"award-info":[{"award-number":["JP18im0210817 JP13bm0104001"]}],"id":[{"id":"10.13039\/100009619","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Physiol."],"abstract":"<jats:p>Duchenne muscular dystrophy (DMD) is an X-linked fatal muscular disease, affecting one in 3,500 live male births worldwide. Currently, there is no cure for this disease, except for steroid-based treatment to attenuate disease progression. Cell transplantation therapy is a promising therapeutic approach, however, there is a lack of appropriate animal models to conduct large-scale preclinical studies using human cells, including biochemical and functional tests. Here, we established an immunodeficient DMD rat model and performed exhaustive pathological analysis and transplantation efficiency evaluation to assess its suitability to study DMD. Our DMD rat model exhibited histopathological characteristics similar to those observed in human patients with DMD. Human myoblasts demonstrated successful engraftment following transplantation into these rats. Therefore, this immunodeficient DMD rat model would be useful in preclinical studies to develop cellular transplantation therapies for DMD.<\/jats:p>","DOI":"10.3389\/fphys.2023.1094359","type":"journal-article","created":{"date-parts":[[2023,4,10]],"date-time":"2023-04-10T04:20:13Z","timestamp":1681100413000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":10,"title":["A new immunodeficient Duchenne muscular dystrophy rat model to evaluate engraftment after human cell transplantation"],"prefix":"10.3389","volume":"14","author":[{"given":"Masae","family":"Sato","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Megumi","family":"Goto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keitaro","family":"Yamanouchi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hidetoshi","family":"Sakurai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2023,4,10]]},"reference":[{"key":"B1","doi-asserted-by":"publisher","first-page":"1611","DOI":"10.1002\/stem.1402","article-title":"A new immuno-dystrophin-deficient model, the NSG-mdx(4Cv) mouse, provides evidence for functional improvement following allogeneic satellite cell transplantation","volume":"31","author":"Arpke","year":"2013","journal-title":"Stem Cells"},{"key":"B2","first-page":"4","article-title":"Drug treatment of duchenne muscular dystrophy: Available evidence and perspectives","volume":"31","author":"Beyt\u00eda","year":"2012","journal-title":"Acta Myol."},{"key":"B3","doi-asserted-by":"crossref","first-page":"223","DOI":"10.1016\/1074-7613(95)90047-0","article-title":"Defective lymphoid development in mice lacking expression of the common cytokine receptor \u03b3 chain","volume":"2","author":"Cao","year":"1995","journal-title":"Immunity"},{"key":"B4","doi-asserted-by":"publisher","first-page":"37","DOI":"10.1016\/j.cell.2008.05.049","article-title":"Highly efficient, functional engraftment of skeletal muscle stem cells in dystrophic muscles","volume":"134","author":"Cerletti","year":"2008","journal-title":"Cell"},{"key":"B5","doi-asserted-by":"crossref","first-page":"2195","DOI":"10.1096\/fj.06-7353com","article-title":"Dystrophin deficient mdx mice display a reduced life span and are susceptible to spontaneous rhabdomyosarcoma","volume":"21","author":"Chamberlain","year":"2007","journal-title":"FASEB J."},{"key":"B6","doi-asserted-by":"crossref","first-page":"128","DOI":"10.1203\/00006450-199207000-00025","article-title":"The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy","volume":"32","author":"Danko","year":"1992","journal-title":"Pediatr. 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