{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T21:46:54Z","timestamp":1790804814773,"version":"4.1.0"},"reference-count":62,"publisher":"Oxford University Press (OUP)","issue":"5","license":[{"start":{"date-parts":[[2024,2,7]],"date-time":"2024-02-07T00:00:00Z","timestamp":1707264000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","award":["W.OR21-01"],"award-info":[{"award-number":["W.OR21-01"]}],"id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004543","name":"China Scholarship Council","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004543","id-type":"DOI","asserted-by":"publisher"}]},{"name":"European Union\u2019s Horizon","award":["101072427"],"award-info":[{"award-number":["101072427"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2024,3,21]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Prime editors have high potential for disease modelling and regenerative medicine efforts including those directed at the muscle-wasting disorder Duchenne muscular dystrophy (DMD). However, the large size and multicomponent nature of prime editing systems pose substantial production and delivery issues. Here, we report that packaging optimized full-length prime editing constructs in adenovector particles (AdVPs) permits installing precise DMD edits in human myogenic cells, namely, myoblasts and mesenchymal stem cells (up to 80% and 64%, respectively). AdVP transductions identified optimized prime-editing reagents capable of correcting\u00a0DMD reading frames of \u223c14% of patient genotypes and restoring\u00a0dystrophin synthesis and dystrophin-\u03b2-dystroglycan linkages in unselected DMD muscle cell populations. AdVPs were equally suitable for correcting DMD iPSC-derived cardiomyocytes and delivering dual prime editors tailored for DMD repair through targeted exon 51 deletion. Moreover, by exploiting the cell cycle-independent AdVP transduction process, we report that 2- and 3-component prime-editing modalities are both most active in cycling than in post-mitotic cells. Finally, we establish that combining AdVP transduction with seamless prime editing allows for stacking chromosomal edits through successive delivery rounds. In conclusion, AdVPs permit versatile investigation of advanced prime editing systems independently of their size and component numbers, which should facilitate their screening and application.<\/jats:p>","DOI":"10.1093\/nar\/gkae057","type":"journal-article","created":{"date-parts":[[2024,2,7]],"date-time":"2024-02-07T09:32:16Z","timestamp":1707298336000},"page":"2740-2757","source":"Crossref","is-referenced-by-count":25,"title":["Selection-free precise gene repair using high-capacity adenovector delivery of advanced prime editing systems rescues dystrophin synthesis in DMD muscle cells"],"prefix":"10.1093","volume":"52","author":[{"given":"Qian","family":"Wang","sequence":"first","affiliation":[{"name":"Leiden University Medical Centre, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC\u00a0 Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabrina","family":"Capelletti","sequence":"additional","affiliation":[{"name":"Leiden University Medical Centre, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC\u00a0 Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin","family":"Liu","sequence":"additional","affiliation":[{"name":"Leiden University Medical Centre, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC\u00a0 Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Josephine M","family":"Janssen","sequence":"additional","affiliation":[{"name":"Leiden University Medical Centre, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC\u00a0 Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8111-1073","authenticated-orcid":false,"given":"Manuel\u00a0A F V","family":"Gon\u00e7alves","sequence":"additional","affiliation":[{"name":"Leiden University Medical Centre, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC\u00a0 Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2024,2,7]]},"reference":[{"key":"2024032102133499100_B1","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"key":"2024032102133499100_B2","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1038\/s41587-021-01133-w","article-title":"Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing","volume":"40","author":"Anzalone","year":"2022","journal-title":"Nat. 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