{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T20:53:56Z","timestamp":1790110436580,"version":"4.0.1"},"reference-count":70,"publisher":"Oxford University Press (OUP)","issue":"13","license":[{"start":{"date-parts":[[2022,7,1]],"date-time":"2022-07-01T00:00:00Z","timestamp":1656633600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"name":"European Union\u2019s Horizon 2020 research and innovation programme","award":["765269"],"award-info":[{"award-number":["765269"]}]},{"DOI":"10.13039\/501100004243","name":"Prinses Beatrix Spierfonds","doi-asserted-by":"publisher","award":["W.OR16-13"],"award-info":[{"award-number":["W.OR16-13"]}],"id":[{"id":"10.13039\/501100004243","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Dutch Duchenne Parent Project","award":["17.012"],"award-info":[{"award-number":["17.012"]}]},{"name":"China Scholarship Council\u2013Leiden University"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2022,7,22]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Targeted chromosomal insertion of large genetic payloads in human cells leverages and broadens synthetic biology and genetic therapy efforts. Yet, obtaining large-scale gene knock-ins remains particularly challenging especially in hard-to-transfect stem and progenitor cells. Here, fully viral gene-deleted adenovector particles (AdVPs) are investigated as sources of optimized high-specificity CRISPR-Cas9 nucleases and donor DNA constructs tailored for targeted insertion of full-length dystrophin expression units (up to 14.8-kb) through homologous recombination (HR) or homology-mediated end joining (HMEJ). In muscle progenitor cells, donors prone to HMEJ yielded higher CRISPR-Cas9-dependent genome editing frequencies than HR donors, with values ranging between 6% and\u00a034%. In contrast, AdVP transduction of HR and HMEJ substrates in induced pluripotent stem cells (iPSCs) resulted in similar CRISPR-Cas9-dependent genome editing levels. Notably, when compared to regular iPSCs, in p53 knockdown iPSCs, CRISPR-Cas9-dependent genome editing frequencies increased up to 6.7-fold specifically when transducing HMEJ donor constructs. Finally, single\u00a0DNA molecule analysis by molecular combing confirmed that AdVP-based genome editing achieves long-term complementation of DMD-causing mutations through the site-specific insertion of full-length dystrophin expression units. In conclusion, AdVPs are a robust and flexible platform for installing large genomic edits in human cells and p53 inhibition fosters HMEJ-based genome editing in iPSCs.<\/jats:p>","DOI":"10.1093\/nar\/gkac567","type":"journal-article","created":{"date-parts":[[2022,7,1]],"date-time":"2022-07-01T14:58:22Z","timestamp":1656687502000},"page":"7761-7782","source":"Crossref","is-referenced-by-count":21,"title":["Large-scale genome editing based on high-capacity adenovectors and\u00a0CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells"],"prefix":"10.1093","volume":"50","author":[{"given":"Francesca","family":"Tasca","sequence":"first","affiliation":[{"name":"Department of Cell and Chemical Biology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC, Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marcella","family":"Brescia","sequence":"additional","affiliation":[{"name":"Department of Cell and Chemical Biology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC, Leiden , The Netherlands"},{"name":"Department of Anatomy and Embryology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC Leiden, The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qian","family":"Wang","sequence":"additional","affiliation":[{"name":"Department of Cell and Chemical Biology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC, Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin","family":"Liu","sequence":"additional","affiliation":[{"name":"Department of Cell and Chemical Biology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC, Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Josephine M","family":"Janssen","sequence":"additional","affiliation":[{"name":"Department of Cell and Chemical Biology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC, Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karoly","family":"Szuhai","sequence":"additional","affiliation":[{"name":"Department of Cell and Chemical Biology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC, Leiden , 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":"Department of Cell and Chemical Biology, Leiden University Medical Center , Einthovenweg 20, 2333 ZC, Leiden , The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2022,7,1]]},"reference":[{"key":"2022072200002462500_B1","doi-asserted-by":"crossref","first-page":"532","DOI":"10.1016\/j.omtm.2020.06.022","article-title":"Ready for repair? gene editing enters the clinic for the treatment of human disease","volume":"18","author":"Ernst","year":"2020","journal-title":"Mol. 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Natl. Acad. Sci. U.S.A."},{"key":"2022072200002462500_B36","doi-asserted-by":"crossref","first-page":"751","DOI":"10.1016\/j.stemcr.2018.01.041","article-title":"Renal subcapsular transplantation of PSC-derived kidney organoids induces neo-vasculogenesis and significant glomerular and tubular maturation in vivo","volume":"10","author":"van\u00a0den\u00a0Berg","year":"2018","journal-title":"Stem Cell Rep."},{"key":"2022072200002462500_B37","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1093\/protein\/13.9.611","article-title":"Functional EGFP-dystrophin fusion proteins for gene therapy vector development","volume":"13","author":"Chapdelaine","year":"2000","journal-title":"Protein. Eng."},{"key":"2022072200002462500_B38","doi-asserted-by":"crossref","first-page":"1331","DOI":"10.1038\/mt.2010.308","article-title":"Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes","volume":"19","author":"Gon\u00e7alves","year":"2011","journal-title":"Mol. Ther."},{"key":"2022072200002462500_B39","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1002\/jgm.2687","article-title":"Development of an adeasy-based system to produce first- and second-generation adenoviral vectors with tropism for CAR- or CD46-positive cells","volume":"15","author":"Janssen","year":"2013","journal-title":"J. Gene Med."},{"key":"2022072200002462500_B40","doi-asserted-by":"crossref","first-page":"18507","DOI":"10.1073\/pnas.0806213105","article-title":"Inducible and reversible gene silencing by stable integration of an shRNA-encoding lentivirus in transgenic rats","volume":"105","author":"Herold","year":"2008","journal-title":"Proc. Natl Acad. Sci. U.S.A."},{"key":"2022072200002462500_B41","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1089\/hum.2012.069","article-title":"Histone deacetylase inhibition activates transgene expression from integration-defective lentiviral vectors in dividing and non-dividing cells","volume":"24","author":"Pelascini","year":"2013","journal-title":"Hum. Gene Ther."},{"key":"2022072200002462500_B42","doi-asserted-by":"crossref","first-page":"181","DOI":"10.1007\/978-1-62703-761-7_12","article-title":"Lentiviral vectors encoding zinc-finger nucleases specific for the model target locus HPRT1","volume":"1114","author":"Pelascini","year":"2014","journal-title":"Methods Mol. Biol."},{"key":"2022072200002462500_B43","doi-asserted-by":"crossref","first-page":"2213","DOI":"10.1038\/s41596-021-00497-2","article-title":"Generation, functional analysis and applications of isogenic three-dimensional self-aggregating cardiac microtissues from human pluripotent stem cells","volume":"16","author":"Campostrini","year":"2021","journal-title":"Nat. Protoc."},{"key":"2022072200002462500_B44","doi-asserted-by":"crossref","first-page":"264","DOI":"10.1038\/nprot.2006.41","article-title":"COBRA: combined binary ratio labeling of nucleic acid probes for multi-color fluorescence in situ hybridization karyotyping","volume":"1","author":"Szuhai","year":"2006","journal-title":"Nat. Protoc."},{"key":"2022072200002462500_B45","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1089\/crispr.2021.0113","article-title":"Inference of CRISPR edits from sanger trace data","volume":"5","author":"Conant","year":"2022","journal-title":"CRISPR J."},{"key":"2022072200002462500_B46","doi-asserted-by":"crossref","first-page":"5105","DOI":"10.1038\/srep05105","article-title":"Adenoviral vector delivery of RNA-guided CRISPR\/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells","volume":"29","author":"Maggio","year":"2014","journal-title":"Sci. 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Cancer Res."},{"key":"2022072200002462500_B53","doi-asserted-by":"crossref","first-page":"167","DOI":"10.1002\/rmv.494","article-title":"Adenovirus: from foe to friend","volume":"16","author":"Gon\u00e7alves","year":"2006","journal-title":"Rev. Med. Virol."},{"key":"2022072200002462500_B54","doi-asserted-by":"crossref","first-page":"1408","DOI":"10.1038\/nm952","article-title":"CD46 is a cellular receptor for group b adenoviruses","volume":"9","author":"Gaggar","year":"2003","journal-title":"Nat. Med."},{"key":"2022072200002462500_B55","doi-asserted-by":"crossref","first-page":"976","DOI":"10.1016\/j.ymthe.2005.11.018","article-title":"Transduction of myogenic cells by retargeted dual high-capacity hybrid viral vectors: robust dystrophin synthesis in duchenne muscular dystrophy muscle cells","volume":"13","author":"Gon\u00e7alves","year":"2006","journal-title":"Mol. Ther."},{"key":"2022072200002462500_B56","doi-asserted-by":"crossref","first-page":"678","DOI":"10.1038\/mt.2016.38","article-title":"Gene insertion into genomic safe harbors for human gene therapy","volume":"24","author":"Papapetrou","year":"2016","journal-title":"Mol. Ther."},{"key":"2022072200002462500_B57","doi-asserted-by":"crossref","first-page":"609650","DOI":"10.3389\/fgeed.2020.609650","article-title":"Targeted gene delivery: where to land","volume":"2","author":"Pavani","year":"2020","journal-title":"Front. Genome Ed."},{"key":"2022072200002462500_B58","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1038\/nmeth.1674","article-title":"Site-specific integration and tailoring of cassette design for sustainable gene transfer","volume":"8","author":"Lombardo","year":"2011","journal-title":"Nat. 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