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Unfortunately, animal models recapitulating such mutations are scarce, limiting our ability to study them and develop genome editing therapies. Here, we describe\u00a0the generation of a novel duplication mouse model, harboring a multi\u2010exonic tandem duplication in the\n                    <jats:italic>Dmd<\/jats:italic>\n                    gene which recapitulates a human mutation. Duplication correction of this mouse was achieved by implementing a single\u2010guide RNA (sgRNA) CRISPR\/Cas9 approach. This strategy precisely removed a duplication mutation\n                    <jats:italic>in vivo<\/jats:italic>\n                    , restored full\u2010length dystrophin expression, and was accompanied by improvements in both histopathological and clinical phenotypes. We conclude that CRISPR\/Cas9 represents a powerful tool to accurately model and treat tandem duplication mutations. Our findings will open new avenues of research for exploring the study and therapeutics of duplication disorders.\n                  <\/jats:p>","DOI":"10.15252\/emmm.202013228","type":"journal-article","created":{"date-parts":[[2021,3,16]],"date-time":"2021-03-16T08:04:12Z","timestamp":1615881852000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":34,"title":["Targeted genome editing in vivo corrects a Dmd duplication restoring wild\u2010type dystrophin expression"],"prefix":"10.1038","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8943-9754","authenticated-orcid":false,"given":"Eleonora","family":"Maino","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daria","family":"Wojtal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonia L","family":"Evagelou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aiman","family":"Farheen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tatianna W Y","family":"Wong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kyle","family":"Lindsay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ori","family":"Scott","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samar Z","family":"Rizvi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elzbieta","family":"Hyatt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Rok","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shagana","family":"Visuvanathan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amanda","family":"Chiodo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle","family":"Schneeweiss","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Evgueni A","family":"Ivakine","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6775-1496","authenticated-orcid":false,"given":"Ronald D","family":"Cohn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,3,16]]},"reference":[{"key":"BFEMMM202013228_CR1","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1086\/381039","volume":"74","author":"A Aartsma\u2010Rus","year":"2004","unstructured":"Aartsma\u2010Rus A, Janson AAM, Kaman WE, Bremmer\u2010Bout M, van Ommen G\u2010JB, den Dunnen JT, van Deutekom JCT (2004) Antisense\u2010induced multiexon skipping for Duchenne muscular dystrophy makes more sense. 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