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Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2022,3]]},"abstract":"<jats:title>Significance<\/jats:title>\n                  <jats:p>\n                    Duchenne muscular dystrophy (DMD) is a fatal disorder of progressive body-wide muscle weakness, considered the most common muscular dystrophy worldwide. Most patients have out-of-frame deletions in the\n                    <jats:italic>DMD<\/jats:italic>\n                    gene, leading to dystrophin absence in muscle. There is no cure for DMD, but exon skipping is emerging as a potential therapy that uses antisense oligonucleotides to convert out-of-frame to in-frame mutations, enabling the production of truncated, partially functional dystrophin. Currently approved exon skipping therapies, however, have limited applicability and efficacy. Here, we developed a more economical approach to skip\n                    <jats:italic>DMD<\/jats:italic>\n                    exons 45 to 55 (a strategy that could treat nearly half of all DMD patients) and identified DG9 peptide conjugation as a powerful way to improve exon skipping efficiencies in\u00a0vivo.\n                  <\/jats:p>","DOI":"10.1073\/pnas.2112546119","type":"journal-article","created":{"date-parts":[[2022,2,22]],"date-time":"2022-02-22T17:11:38Z","timestamp":1645549898000},"update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":50,"title":["Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy"],"prefix":"10.1073","volume":"119","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5484-4183","authenticated-orcid":false,"given":"Kenji Rowel Q.","family":"Lim","sequence":"first","affiliation":[{"name":"Department of Medical Genetics, Faculty of 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