{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,26]],"date-time":"2026-02-26T17:20:41Z","timestamp":1772126441840,"version":"3.50.1"},"reference-count":34,"publisher":"Public Library of Science (PLoS)","issue":"4","license":[{"start":{"date-parts":[[2022,4,4]],"date-time":"2022-04-04T00:00:00Z","timestamp":1649030400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100009619","name":"Japan Agency for Medical Research and Development","doi-asserted-by":"publisher","award":["JP21bm0104001"],"award-info":[{"award-number":["JP21bm0104001"]}],"id":[{"id":"10.13039\/100009619","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:p>Duchenne muscular dystrophy (DMD) is an intractable genetic muscular disorder characterized by the loss of DYSTROPHIN. The restoration of DYSTROPHIN is expected to be a curative therapy for DMD. Because muscle stem cells (MuSCs) can regenerate damaged myofibers with full-length DYSTROPHIN<jats:italic>in vivo<\/jats:italic>, their transplantation is being explored as such a therapy. As for the transplanted cells, primary satellite cells have been considered, but donor shortage limits their clinical application. We previously developed a protocol that differentiates induced pluripotent stem cells (iPSCs) to MuSCs (iMuSCs). To ameliorate the respiratory function of DMD patients, cell transplantation to the diaphragm is necessary but difficult, because the diaphragm is thin and rapidly moves. In the present study, we explored the transplantation of iMuSCs into the diaphragm. First, we show direct cell injection into the diaphragm of mouse was feasible. Then, to enhance the engraftment of the transplanted cells in a rapidly moving diaphragm, we mixed polymer solutions of hyaluronic acid, alginate and gelatin to the cell suspension, finding a solution of 20% dissolved hyaluronic acid and 80% dissolved gelatin improved the engraftment. Thus, we established a method for cell transplantation into mouse diaphragm and show that an injectable hyaluronic acid-gelatin solution enables the engraftment of iMuSCs in the diaphragm.<\/jats:p>","DOI":"10.1371\/journal.pone.0266391","type":"journal-article","created":{"date-parts":[[2022,4,4]],"date-time":"2022-04-04T17:30:16Z","timestamp":1649093416000},"page":"e0266391","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":18,"title":["Transplantation of human iPSC-derived muscle stem cells in the diaphragm of Duchenne muscular dystrophy model mice"],"prefix":"10.1371","volume":"17","author":[{"given":"Yasutomo","family":"Miura","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mase","family":"Sato","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Toshie","family":"Kuwahara","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tomoki","family":"Ebata","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yasuhiko","family":"Tabata","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5383-9366","authenticated-orcid":true,"given":"Hidetoshi","family":"Sakurai","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"340","published-online":{"date-parts":[[2022,4,4]]},"reference":[{"issue":"4","key":"pone.0266391.ref001","doi-asserted-by":"crossref","first-page":"reviews3006.1","DOI":"10.1186\/gb-2001-2-4-reviews3006","article-title":"Dystrophins and dystrobrevins.","volume":"2","author":"RG Roberts","year":"2001","journal-title":"Genome Biology"},{"issue":"6","key":"pone.0266391.ref002","doi-asserted-by":"crossref","first-page":"373","DOI":"10.1038\/nrg3460","article-title":"Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches","volume":"14","author":"RJ Fairclough","year":"2013","journal-title":"Nat Rev Genet"},{"issue":"2","key":"pone.0266391.ref003","first-page":"121","article-title":"Improvement of survival in Duchenne Muscular Dystrophy: retrospective analysis of 835 patients.","volume":"31","author":"L Passamano","year":"2012","journal-title":"Acta Myol."},{"issue":"10119","key":"pone.0266391.ref004","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1016\/S0140-6736(17)32160-8","article-title":"Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study","volume":"391","author":"CM McDonald","year":"2018","journal-title":"Lancet"},{"issue":"10","key":"pone.0266391.ref005","doi-asserted-by":"crossref","first-page":"918","DOI":"10.1016\/S1474-4422(09)70211-X","article-title":"Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study","volume":"8","author":"M Kinali","year":"2009","journal-title":"Lancet Neurol"},{"issue":"R2","key":"pone.0266391.ref006","doi-asserted-by":"crossref","first-page":"R163","DOI":"10.1093\/hmg\/ddy171","article-title":"Exon-skipping advances for Duchenne muscular dystrophy","volume":"27","author":"L Echevarr\u00eda","year":"2018","journal-title":"Hum Mol Genet"},{"issue":"10","key":"pone.0266391.ref007","doi-asserted-by":"crossref","first-page":"2337","DOI":"10.1016\/j.ymthe.2018.07.011","article-title":"Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy","volume":"26","author":"D. 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