{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T22:40:02Z","timestamp":1792795202157,"version":"4.5.0"},"reference-count":35,"publisher":"American Association for the Advancement of Science (AAAS)","issue":"6410","funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["HL130253, HL077439, DK099653, AR067294 and U54HD087351"],"award-info":[{"award-number":["HL130253, HL077439, DK099653, AR067294 and U54HD087351"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000928","name":"Welch Foundation","doi-asserted-by":"publisher","award":["1-0025"],"award-info":[{"award-number":["1-0025"]}],"id":[{"id":"10.13039\/100000928","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Science"],"published-print":{"date-parts":[[2018,10,5]]},"abstract":"<jats:title>Gene editing and muscular dystrophy<\/jats:title>\n                  <jats:p>\n                    Duchenne muscular dystrophy (DMD) is characterized by progressive muscle weakness and a shortened life span. The disease is caused by mutations that reduce or prevent expression of dystrophin, an essential structural protein in skeletal and heart muscle. The gene editing technology CRISPR-Cas9 can correct disease-causing mutations and has yielded promising results in mouse models of DMD. In a small, short-term study, Amoasii\n                    <jats:italic>et al.<\/jats:italic>\n                    tested this strategy in a dog model of DMD that exhibits many features of the human disease. Intramuscular or systemic delivery of the gene editing components resulted in a substantial increase in dystrophin protein levels in skeletal and heart muscle. Restoration of dystrophin expression was accompanied by improved muscle histology.\n                  <\/jats:p>\n                  <jats:p>\n                    <jats:italic>Science<\/jats:italic>\n                    , this issue p.\n                    <jats:related-article xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"doi\" issue=\"6410\" page=\"86\" related-article-type=\"in-this-issue\" vol=\"362\" xlink:href=\"10.1126\/science.aau1549\">86<\/jats:related-article>\n                  <\/jats:p>","DOI":"10.1126\/science.aau1549","type":"journal-article","created":{"date-parts":[[2018,8,30]],"date-time":"2018-08-30T14:07:12Z","timestamp":1535638032000},"page":"86-91","source":"Crossref","is-referenced-by-count":501,"title":["Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy"],"prefix":"10.1126","volume":"362","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7960-2749","authenticated-orcid":true,"given":"Leonela","family":"Amoasii","sequence":"first","affiliation":[{"name":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA."},{"name":"Exonics Therapeutics, 75 Kneeland Street, Boston, MA 02111, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2283-2118","authenticated-orcid":true,"given":"John C. W.","family":"Hildyard","sequence":"additional","affiliation":[{"name":"Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4114-8349","authenticated-orcid":true,"given":"Hui","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9358-1630","authenticated-orcid":true,"given":"Efrain","family":"Sanchez-Ortiz","sequence":"additional","affiliation":[{"name":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7534-636X","authenticated-orcid":true,"given":"Alex","family":"Mireault","sequence":"additional","affiliation":[{"name":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. Paul D. 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Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9418-0708","authenticated-orcid":true,"given":"Rachel","family":"Harron","sequence":"additional","affiliation":[{"name":"Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4666-2002","authenticated-orcid":true,"given":"Thaleia-Rengina","family":"Stathopoulou","sequence":"additional","affiliation":[{"name":"Section of Anaesthesia and Analgesia, Royal Veterinary College, London NW1 0TU, UK."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0823-6547","authenticated-orcid":true,"given":"Claire","family":"Massey","sequence":"additional","affiliation":[{"name":"Department of Clinical Science and Services, Comparative Neuromuscular Diseases Laboratory, Royal Veterinary College, London NW1 0TU, UK."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2949-1638","authenticated-orcid":true,"given":"John M.","family":"Shelton","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7568-410X","authenticated-orcid":true,"given":"Rhonda","family":"Bassel-Duby","sequence":"additional","affiliation":[{"name":"Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Sen. 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