{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T03:21:58Z","timestamp":1793244118478,"version":"4.5.0"},"reference-count":35,"publisher":"American Society for Clinical Investigation","issue":"7","funder":[{"name":"Coalition Duchenne","award":["N\/A"],"award-info":[{"award-number":["N\/A"]}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["T32 HL116273"],"award-info":[{"award-number":["T32 HL116273"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["R01 HL124074"],"award-info":[{"award-number":["R01 HL124074"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2019,4,4]]},"abstract":"<jats:p>\n                    Dystrophin deficiency leads to progressive muscle degeneration in Duchenne muscular dystrophy (DMD) patients. No known cure exists, and standard care relies on the use of antiinflammatory steroids, which are associated with side effects that complicate long-term use. Here, we report that a single intravenous dose of clinical-stage cardiac stromal cells, called cardiosphere-derived cells (CDCs), improves the dystrophic phenotype in\n                    <jats:italic>mdx<\/jats:italic>\n                    mice. CDCs augment cardiac and skeletal muscle function, partially reverse established heart damage, and boost the regenerative capacity of skeletal muscle. We further demonstrate that CDCs work by secreting exosomes, which normalize gene expression at the transcriptome level, and alter cell signaling and biological processes in\n                    <jats:italic>mdx<\/jats:italic>\n                    hearts and skeletal muscle. The work reported here motivated the ongoing HOPE-2 clinical trial of systemic CDC delivery to DMD patients, and identifies exosomes as next-generation cell-free therapeutic candidates for DMD.\n                  <\/jats:p>","DOI":"10.1172\/jci.insight.125754","type":"journal-article","created":{"date-parts":[[2019,4,3]],"date-time":"2019-04-03T13:41:59Z","timestamp":1554298919000},"source":"Crossref","is-referenced-by-count":64,"title":["Disease-modifying bioactivity of intravenous cardiosphere-derived cells and exosomes in mdx mice"],"prefix":"10.1172","volume":"4","author":[{"given":"Russell G.","family":"Rogers","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mario","family":"Fournier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lizbeth","family":"Sanchez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ahmed G.","family":"Ibrahim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark A.","family":"Aminzadeh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael I.","family":"Lewis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eduardo","family":"Marb\u00e1n","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"232","published-online":{"date-parts":[[2019,4,4]]},"reference":[{"issue":"4","key":"B1","first-page":"498","article-title":"The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion","volume":"45","author":"Koenig","year":"1989","journal-title":"Am J Hum Genet"},{"key":"B2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70272-8"},{"key":"B3","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.05.019"},{"key":"B4","doi-asserted-by":"publisher","DOI":"10.1038\/s41551-018-0216-z"},{"key":"B5","doi-asserted-by":"crossref","unstructured":"Aminzadeh MA, et al. Reversal of cardiac and skeletal manifestations of Duchenne muscular dystrophy by cardiosphere-derived cells and their exosomes in mdx dystrophic mice and in human Duchenne cardiomyocytes. bioRxiv Web site. https:\/\/www.biorxiv.org\/content\/10.1101\/128900v1 Uploaded April 20, 2017. Access March 1, 2019","DOI":"10.1101\/128900"},{"key":"B6","doi-asserted-by":"publisher","DOI":"10.1016\/j.stemcr.2014.04.006"},{"key":"B7","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-physiol-021115-104929"},{"key":"B8","doi-asserted-by":"crossref","unstructured":"Rogers RG, et al. Intravenous delivery of cardiosphere-derived cells improves striated muscle function and structure in a murine model of Duchenne muscular dystrophy. FASEB J. 2017;31(suppl 1):877.10\u2013877.10. https:\/\/www.fasebj.org\/doi\/abs\/10.1096\/fasebj.31.1_supplement.877.10","DOI":"10.1096\/fasebj.31.1_supplement.877.10"},{"key":"B9","unstructured":"Rogers RG, Fournier M, Aminzadeh MA, Gouin K, Sanchez L, and Marb\u00e1n E. Abstract 16576: Intravenous infusion of cardiosphere-derived cells and their exosomes improve dystrophin-deficient cardiomyopathy in mdx mice. 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