{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T23:29:16Z","timestamp":1787009356000,"version":"build-2736575974"},"reference-count":70,"publisher":"Oxford University Press (OUP)","issue":"2","license":[{"start":{"date-parts":[[2024,12,9]],"date-time":"2024-12-09T00:00:00Z","timestamp":1733702400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004543","name":"China Scholarship Council","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004543","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Dutch Duchenne Parent"},{"DOI":"10.13039\/100018693","name":"Horizon Europe","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018693","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003246","name":"Dutch Research Council","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003246","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,1,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Genome editing based on programmable nucleases and donor DNA constructs permits introducing specific base-pair changes and complete transgenes or live-cell reporter tags at predefined chromosomal positions. A crucial requirement for such versatile genome editing approaches is, however, the need to co-deliver in an effective, coordinated and non-cytotoxic manner all the required components into target cells. Here, adenoviral (AdV) and adeno-associated viral (AAV) vectors are investigated as delivery agents for, respectively, engineered CRISPR-Cas9 nucleases and donor DNA constructs prone to homologous recombination (HR) or homology-mediated end joining (HMEJ) processes. Specifically, canonical single-stranded and self-complementary double-stranded AAVs served as sources of ectopic HR and HMEJ substrates, whilst second- and third-generation AdVs provided for matched CRISPR-Cas9 nucleases. We report that combining single-stranded AAV delivery of HR donors with third-generation AdV transfer of CRISPR-Cas9 nucleases results in selection-free and precise whole transgene insertion in large fractions of target-cell populations (i.e. up to 93%) and disclose that programmable nuclease-induced chromosomal breaks promote AAV transduction. Finally, besides investigating relationships between distinct AAV structures and genome-editing performance endpoints, we further report that high-fidelity CRISPR-Cas9 nucleases are critical for mitigating off-target chromosomal insertion of defective AAV genomes known to be packaged in vector particles.<\/jats:p>","DOI":"10.1093\/nar\/gkae1213","type":"journal-article","created":{"date-parts":[[2024,11,22]],"date-time":"2024-11-22T07:28:19Z","timestamp":1732260499000},"source":"Crossref","is-referenced-by-count":13,"title":["Precision genome editing using combinatorial viral vector delivery of CRISPR-Cas9 nucleases and donor DNA constructs"],"prefix":"10.1093","volume":"53","author":[{"given":"Zhen","family":"Li","sequence":"first","affiliation":[{"name":"Leiden University Medical Center, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC Leiden ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaoling","family":"Wang","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC Leiden ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Josephine M","family":"Janssen","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC Leiden ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin","family":"Liu","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC Leiden ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Tasca","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC Leiden ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rob C","family":"Hoeben","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC Leiden ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8111-1073","authenticated-orcid":false,"given":"Manuel A F V","family":"Gon\u00e7alves","sequence":"additional","affiliation":[{"name":"Leiden University Medical Center, Department of Cell and Chemical Biology , Einthovenweg 20, 2333 ZC Leiden ,","place":["the Netherlands"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2024,12,9]]},"reference":[{"key":"2025012307151453500_B1","doi-asserted-by":"crossref","first-page":"1076","DOI":"10.1016\/j.cell.2024.01.042","article-title":"Past, present, and future of CRISPR genome editing technologies","volume":"187","author":"Pacesa","year":"2024","journal-title":"Cell"},{"key":"2025012307151453500_B2","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1016\/j.isci.2018.08.001","article-title":"DNA, RNA, and protein tools for editing the genetic information in Human cells","volume":"6","author":"Chen","year":"2018","journal-title":"iScience"},{"key":"2025012307151453500_B3","doi-asserted-by":"crossref","first-page":"102344","DOI":"10.1016\/j.omtn.2024.102344","article-title":"CRISPR\/Cas9-mediated homology-directed repair for precise gene editing","volume":"35","author":"Liao","year":"2024","journal-title":"Mol. 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