{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T20:14:17Z","timestamp":1791231257052,"version":"4.1.0"},"reference-count":83,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2022,10,28]],"date-time":"2022-10-28T00:00:00Z","timestamp":1666915200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004337","name":"Roche","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004337","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2022,12]]},"DOI":"10.1016\/j.omtm.2022.10.016","type":"journal-article","created":{"date-parts":[[2022,11,1]],"date-time":"2022-11-01T13:11:24Z","timestamp":1667308284000},"page":"431-449","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":16,"special_numbering":"C","title":["Population-wide gene disruption in the murine lung epithelium via AAV-mediated delivery of CRISPR-Cas9 components"],"prefix":"10.1016","volume":"27","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0261-3033","authenticated-orcid":false,"given":"Honglin","family":"Chen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steffen","family":"Durinck","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hetal","family":"Patel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Oded","family":"Foreman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathryn","family":"Mesh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Eastham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roger","family":"Caothien","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert J.","family":"Newman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Merone","family":"Roose-Girma","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Spyros","family":"Darmanis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Soren","family":"Warming","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Annalisa","family":"Lattanzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1143-423X","authenticated-orcid":false,"given":"Yuxin","family":"Liang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0074-0020","authenticated-orcid":false,"given":"Benjamin","family":"Haley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2022.10.016_bib1","doi-asserted-by":"crossref","first-page":"11854","DOI":"10.1073\/pnas.182412299","article-title":"Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy","volume":"99","author":"Gao","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2022.10.016_bib2","doi-asserted-by":"crossref","first-page":"6381","DOI":"10.1128\/JVI.78.12.6381-6388.2004","article-title":"Clades of adeno-associated viruses are widely disseminated in human tissues","volume":"78","author":"Gao","year":"2004","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2022.10.016_bib3","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1016\/j.ymthe.2006.03.014","article-title":"Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8","volume":"14","author":"Inagaki","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib4","doi-asserted-by":"crossref","first-page":"e3","DOI":"10.1161\/01.RES.0000237661.18885.f6","article-title":"Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in\u00a0vivo","volume":"99","author":"Pacak","year":"2006","journal-title":"Circ. Res."},{"key":"10.1016\/j.omtm.2022.10.016_bib5","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1038\/gt.2017.19","article-title":"Optimization of adeno-associated virus vector-mediated gene transfer to the respiratory tract","volume":"24","author":"Kurosaki","year":"2017","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib6","doi-asserted-by":"crossref","first-page":"528","DOI":"10.1016\/j.ymthe.2005.11.015","article-title":"Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain","volume":"13","author":"Cearley","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib7","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1016\/j.ymthe.2004.05.024","article-title":"Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system","volume":"10","author":"Burger","year":"2004","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib8","doi-asserted-by":"crossref","first-page":"1042","DOI":"10.1038\/mt.2016.61","article-title":"Superior in\u00a0vivo transduction of human hepatocytes using engineered AAV3 capsid","volume":"24","author":"Vercauteren","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib9","doi-asserted-by":"crossref","first-page":"294","DOI":"10.1038\/mt.2008.261","article-title":"Transduction efficiencies of novel AAV vectors in mouse airway epithelium in\u00a0vivo and human ciliated airway epithelium in\u00a0vitro","volume":"17","author":"Limberis","year":"2009","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib10","doi-asserted-by":"crossref","first-page":"12993","DOI":"10.1073\/pnas.0601433103","article-title":"Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered","volume":"103","author":"Limberis","year":"2006","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2022.10.016_bib11","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2016.42","article-title":"Multilineage transduction of resident lung cells in\u00a0vivo by AAV2\/8 for \u03b11-antitrypsin gene therapy","volume":"3","author":"Payne","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2022.10.016_bib12","doi-asserted-by":"crossref","first-page":"939","DOI":"10.1158\/1078-0432.CCR-07-1930","article-title":"Suppression of lung tumor growth and metastasis in mice by adeno-associated virus-mediated expression of vasostatin","volume":"14","author":"Cai","year":"2008","journal-title":"Clin. Cancer Res."},{"key":"10.1016\/j.omtm.2022.10.016_bib13","doi-asserted-by":"crossref","DOI":"10.3389\/fonc.2021.667189","article-title":"SEMMs: somatically engineered mouse models. A new tool for in\u00a0vivo disease modeling for basic and translational research","volume":"11","author":"Lima","year":"2021","journal-title":"Front. Oncol."},{"key":"10.1016\/j.omtm.2022.10.016_bib14","doi-asserted-by":"crossref","first-page":"423","DOI":"10.1038\/nature13902","article-title":"In\u00a0vivo engineering of oncogenic chromosomal rearrangements with the CRISPR\/Cas9 system","volume":"516","author":"Maddalo","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2022.10.016_bib15","doi-asserted-by":"crossref","first-page":"1219","DOI":"10.1016\/j.celrep.2014.10.051","article-title":"Simple and rapid in\u00a0vivo generation of chromosomal rearrangements using CRISPR\/Cas9 technology","volume":"9","author":"Blasco","year":"2014","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtm.2022.10.016_bib16","doi-asserted-by":"crossref","first-page":"440","DOI":"10.1016\/j.cell.2014.09.014","article-title":"CRISPR-Cas9 knockin mice for genome editing and cancer modeling","volume":"159","author":"Platt","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2022.10.016_bib17","doi-asserted-by":"crossref","first-page":"428","DOI":"10.1038\/nature13906","article-title":"Rapid modeling of cooperating genetic events in cancer through somatic genome editing","volume":"516","author":"S\u00e1nchez-Rivera","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2022.10.016_bib18","doi-asserted-by":"crossref","first-page":"3214","DOI":"10.1158\/2159-8290.CD-21-0441","article-title":"Rlf-Mycl gene fusion drives tumorigenesis and metastasis in a mouse model of small cell lung cancer","volume":"11","author":"Ciampricotti","year":"2021","journal-title":"Cancer Discov."},{"key":"10.1016\/j.omtm.2022.10.016_bib19","doi-asserted-by":"crossref","DOI":"10.3389\/fcell.2021.641618","article-title":"Implementation of CRISPR\/Cas9 genome editing to generate murine lung cancer models that depict the mutational landscape of human disease","volume":"9","author":"Hartmann","year":"2021","journal-title":"Front. Cell Dev. Biol."},{"key":"10.1016\/j.omtm.2022.10.016_bib20","doi-asserted-by":"crossref","DOI":"10.1038\/ncomms15987","article-title":"Somatic chromosomal engineering identifies BCAN-NTRK1 as a potent glioma driver and therapeutic target","volume":"8","author":"Cook","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2022.10.016_bib21","doi-asserted-by":"crossref","first-page":"1233","DOI":"10.1038\/s41374-018-0171-z","article-title":"A pipeline for rapidly generating genetically engineered mouse models of pancreatic cancer using in\u00a0vivo CRISPR-Cas9-mediated somatic recombination","volume":"99","author":"Ideno","year":"2019","journal-title":"Lab. Invest."},{"key":"10.1016\/j.omtm.2022.10.016_bib22","doi-asserted-by":"crossref","DOI":"10.15252\/embj.2019102169","article-title":"In situ CRISPR-Cas9 base editing for the development of genetically engineered mouse models of breast cancer","volume":"39","author":"Annunziato","year":"2020","journal-title":"EMBO J."},{"key":"10.1016\/j.omtm.2022.10.016_bib23","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/nprot.2017.136","article-title":"Colonoscopy-based colorectal cancer modeling in mice with CRISPR\u2013Cas9 genome editing and organoid transplantation","volume":"13","author":"Roper","year":"2018","journal-title":"Nat. Protoc."},{"key":"10.1016\/j.omtm.2022.10.016_bib24","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1016\/j.omtn.2022.07.023","article-title":"Effective splicing restoration of a deep-intronic ABCA4 variant in cone photoreceptor precursor cells by CRISPR\/SpCas9 approaches","volume":"29","author":"De Angeli","year":"2022","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2022.10.016_bib25","article-title":"Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system","volume":"6","author":"Zhang","year":"2020","journal-title":"Sci. Adv."},{"key":"10.1016\/j.omtm.2022.10.016_bib26","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1126\/science.aad5177","article-title":"In\u00a0vivo gene editing in dystrophic mouse muscle and muscle stem cells","volume":"351","author":"Tabebordbar","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.omtm.2022.10.016_bib27","doi-asserted-by":"crossref","first-page":"646","DOI":"10.1038\/s41434-021-00224-2","article-title":"Highly efficient neuronal gene knockout in\u00a0vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice","volume":"28","author":"Hana","year":"2021","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib28","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1016\/j.jcf.2010.03.001","article-title":"Alveolar inflammation in cystic fibrosis","volume":"9","author":"Ulrich","year":"2010","journal-title":"J.\u00a0Cyst. Fibros."},{"key":"10.1016\/j.omtm.2022.10.016_bib29","doi-asserted-by":"crossref","first-page":"2269","DOI":"10.3390\/ijms21072269","article-title":"Alveolar epithelial type II cells as drivers of lung fibrosis in idiopathic pulmonary fibrosis","volume":"21","author":"Parimon","year":"2020","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.omtm.2022.10.016_bib30","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0237529","article-title":"Dysregulation of club cell biology in idiopathic pulmonary fibrosis","volume":"15","author":"Zuo","year":"2020","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2022.10.016_bib31","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1186\/s12950-017-0168-1","article-title":"Depletion of club cells attenuates bleomycin-induced lung injury and fibrosis in mice","volume":"14","author":"Yokoyama","year":"2017","journal-title":"J.\u00a0Inflamm."},{"key":"10.1016\/j.omtm.2022.10.016_bib32","doi-asserted-by":"crossref","first-page":"133","DOI":"10.1038\/nn.2467","article-title":"A robust and high-throughput Cre reporting and characterization system for the whole mouse brain","volume":"13","author":"Madisen","year":"2010","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.omtm.2022.10.016_bib33","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1038\/s41591-019-0344-3","article-title":"Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy","volume":"25","author":"Nelson","year":"2019","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2022.10.016_bib34","doi-asserted-by":"crossref","first-page":"4439","DOI":"10.1038\/s41467-019-12449-2","article-title":"High levels of AAV vector integration into CRISPR-induced DNA breaks","volume":"10","author":"Hanlon","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2022.10.016_bib35","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1186\/s12864-020-6655-4","article-title":"ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in\u00a0vivo following adeno-associated viral vector-mediated genome editing","volume":"21","author":"Breton","year":"2020","journal-title":"BMC Genom."},{"key":"10.1016\/j.omtm.2022.10.016_bib36","doi-asserted-by":"crossref","first-page":"344","DOI":"10.1089\/hum.2006.182","article-title":"High-efficiency promoter-dependent transduction by adeno-associated virus type 6 vectors in mouse lung","volume":"18","author":"Halbert","year":"2007","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib37","doi-asserted-by":"crossref","first-page":"1994","DOI":"10.1016\/j.ymthe.2022.01.010","article-title":"Non-invasive administration of AAV to target lung parenchymal cells and develop SARS-CoV-2-susceptible mice","volume":"30","author":"Yang","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib38","doi-asserted-by":"crossref","first-page":"1073","DOI":"10.1038\/mt.2008.76","article-title":"Analysis of AAV serotypes 1\u20139 mediated gene expression and tropism in mice after systemic injection","volume":"16","author":"Zincarelli","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib39","doi-asserted-by":"crossref","first-page":"186","DOI":"10.1038\/nature14299","article-title":"In\u00a0vivo genome editing using Staphylococcus aureus Cas9","volume":"520","author":"Ran","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2022.10.016_bib40","doi-asserted-by":"crossref","first-page":"3903","DOI":"10.1073\/pnas.1621177114","article-title":"Transcription factor Etv5 is essential for the maintenance of alveolar type II cells","volume":"114","author":"Zhang","year":"2017","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2022.10.016_bib41","doi-asserted-by":"crossref","first-page":"2855","DOI":"10.1172\/JCI57673","article-title":"Integrin \u03b16\u03b24 identifies an adult distal lung epithelial population with regenerative potential in mice","volume":"121","author":"Chapman","year":"2011","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2022.10.016_bib42","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1038\/nature14112","article-title":"Lineage-negative progenitors mobilize to regenerate lung epithelium after major injury","volume":"517","author":"Vaughan","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2022.10.016_bib43","doi-asserted-by":"crossref","first-page":"868","DOI":"10.1038\/nmeth.3993","article-title":"A multifunctional AAV\u2013CRISPR\u2013Cas9 and its host response","volume":"13","author":"Chew","year":"2016","journal-title":"Nat. Methods"},{"key":"10.1016\/j.omtm.2022.10.016_bib44","doi-asserted-by":"crossref","first-page":"6450","DOI":"10.1093\/nar\/gkv601","article-title":"Development of an intein-mediated split\u2013Cas9 system for gene therapy","volume":"43","author":"Truong","year":"2015","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtm.2022.10.016_bib45","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1186\/1756-6606-7-17","article-title":"Optimization of AAV expression cassettes to improve packaging capacity and transgene expression in neurons","volume":"7","author":"Choi","year":"2014","journal-title":"Mol. Brain"},{"key":"10.1016\/j.omtm.2022.10.016_bib46","doi-asserted-by":"crossref","first-page":"238","DOI":"10.1016\/j.ymthe.2021.10.023","article-title":"AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway","volume":"30","author":"Liang","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib47","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1038\/nbt.3149","article-title":"A split-Cas9 architecture for inducible genome editing and transcription modulation","volume":"33","author":"Zetsche","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2022.10.016_bib48","doi-asserted-by":"crossref","first-page":"2984","DOI":"10.1073\/pnas.1501698112","article-title":"Rational design of a split-Cas9 enzyme complex","volume":"112","author":"Wright","year":"2015","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2022.10.016_bib49","doi-asserted-by":"crossref","first-page":"370","DOI":"10.1038\/nature13418","article-title":"The mitochondrial deubiquitinase USP30 opposes parkin-mediated mitophagy","volume":"510","author":"Bingol","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2022.10.016_bib50","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1083\/jcb.201603039","article-title":"mito-QC illuminates mitophagy and mitochondrial architecture in\u00a0vivo","volume":"214","author":"McWilliams","year":"2016","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.omtm.2022.10.016_bib51","doi-asserted-by":"crossref","first-page":"4365","DOI":"10.1242\/dev.083840","article-title":"Different assemblies of Notch receptors coordinate the distribution of the major bronchial Clara, ciliated and neuroendocrine cells","volume":"139","author":"Morimoto","year":"2012","journal-title":"Development"},{"key":"10.1016\/j.omtm.2022.10.016_bib52","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1038\/nature15715","article-title":"Therapeutic antibodies reveal Notch control of transdifferentiation in the adult lung","volume":"528","author":"Lafkas","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2022.10.016_bib53","doi-asserted-by":"crossref","first-page":"1524","DOI":"10.1128\/JVI.74.3.1524-1532.2000","article-title":"Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes","volume":"74","author":"Halbert","year":"2000","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2022.10.016_bib54","doi-asserted-by":"crossref","first-page":"2646","DOI":"10.1016\/j.ymthe.2022.06.004","article-title":"Evaluating the state of the science for adeno-associated virus integration: an integrated perspective","volume":"30","author":"Sabatino","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib55","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1038\/s41587-020-0741-7","article-title":"A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells","volume":"39","author":"Nguyen","year":"2021","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2022.10.016_bib56","doi-asserted-by":"crossref","first-page":"1671","DOI":"10.1002\/humu.24063","article-title":"Xdrop: targeted sequencing of long DNA molecules from low input samples using droplet sorting","volume":"41","author":"Madsen","year":"2020","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.omtm.2022.10.016_bib57","doi-asserted-by":"crossref","first-page":"2898","DOI":"10.1016\/j.ymthe.2021.08.031","article-title":"AAV integration in human hepatocytes","volume":"29","author":"Dalwadi","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib58","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1586\/1744666X.5.1.63","article-title":"Role of the pulmonary epithelium and inflammatory signals in acute lung injury","volume":"5","author":"Manicone","year":"2014","journal-title":"Expert Rev. Clin. Immunol."},{"key":"10.1016\/j.omtm.2022.10.016_bib59","doi-asserted-by":"crossref","first-page":"686","DOI":"10.1038\/nri3739","article-title":"The role of airway epithelial cells and innate immune cells in chronic respiratory disease","volume":"14","author":"Holtzman","year":"2014","journal-title":"Nat. Rev. Immunol."},{"key":"10.1016\/j.omtm.2022.10.016_bib60","article-title":"In\u00a0vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni","volume":"8","author":"Kim","year":"2017","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2022.10.016_bib61","doi-asserted-by":"crossref","first-page":"4219","DOI":"10.1038\/s41467-021-24454-5","article-title":"Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases","volume":"12","author":"Schmidt","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2022.10.016_bib62","doi-asserted-by":"crossref","first-page":"3929","DOI":"10.1038\/s41467-020-17577-8","article-title":"A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency","volume":"11","author":"Kang","year":"2020","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2022.10.016_bib63","doi-asserted-by":"crossref","first-page":"323","DOI":"10.1016\/j.omtm.2018.04.005","article-title":"A novel triple-mutant AAV6 capsid induces rapid and potent transgene expression in the muscle and respiratory tract of mice","volume":"9","author":"van Lieshout","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2022.10.016_bib64","doi-asserted-by":"crossref","first-page":"9795","DOI":"10.1128\/JVI.72.12.9795-9805.1998","article-title":"Successful readministration of adeno-associated virus vectors to the mouse lung requires transient immunosuppression during the initial exposure","volume":"72","author":"Halbert","year":"1998","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.omtm.2022.10.016_bib65","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1158\/2159-8290.CD-16-0665","article-title":"Genomic amplifications cause false positives in CRISPR screens","volume":"6","author":"Sheel","year":"2016","journal-title":"Cancer Discov."},{"key":"10.1016\/j.omtm.2022.10.016_bib66","doi-asserted-by":"crossref","first-page":"390","DOI":"10.1038\/nbt.3155","article-title":"Inducible in\u00a0vivo genome editing with CRISPR\/Cas9","volume":"33","author":"Dow","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2022.10.016_bib67","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1038\/nchembio.1793","article-title":"Small molecule-triggered Cas9 protein with improved genome-editing specificity","volume":"11","author":"Davis","year":"2015","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.omtm.2022.10.016_bib68","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1002\/cbic.202000423","article-title":"Regulating CRISPR\/Cas9 function through conditional guide RNA control","volume":"22","author":"Brown","year":"2021","journal-title":"Chembiochem"},{"key":"10.1016\/j.omtm.2022.10.016_bib69","doi-asserted-by":"crossref","first-page":"1216","DOI":"10.1038\/s41591-018-0137-0","article-title":"A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells","volume":"24","author":"Vakulskas","year":"2018","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtm.2022.10.016_bib70","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1038\/s41551-019-0501-5","article-title":"Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses","volume":"4","author":"Levy","year":"2020","journal-title":"Nat. Biomed. Eng."},{"key":"10.1016\/j.omtm.2022.10.016_bib71","doi-asserted-by":"crossref","first-page":"eabf2444","DOI":"10.1126\/scitranslmed.abf2444","article-title":"Development of \u03b2-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease","volume":"13","author":"Lattanzi","year":"2021","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2022.10.016_bib72","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/j.ymthe.2016.12.006","article-title":"CRISPR\/Cas9-Mediated genome editing as a therapeutic approach for leber congenital Amaurosis 10","volume":"25","author":"Ruan","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib73","doi-asserted-by":"crossref","first-page":"571","DOI":"10.1089\/hum.2018.193","article-title":"Somatic gene editing of GUCY2D by AAV-CRISPR\/Cas9 alters retinal structure and function in mouse and macaque","volume":"30","author":"McCullough","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2022.10.016_bib74","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1126\/science.aad5143","article-title":"In\u00a0vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy","volume":"351","author":"Nelson","year":"2016","journal-title":"Science"},{"key":"10.1016\/j.omtm.2022.10.016_bib75","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1016\/j.omtn.2017.02.004","article-title":"Correction of the exon 2 duplication in DMD myoblasts by a single CRISPR\/Cas9 system","volume":"7","author":"Lattanzi","year":"2017","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtm.2022.10.016_bib76","doi-asserted-by":"crossref","first-page":"798","DOI":"10.1186\/s12864-018-5164-1","article-title":"Correcting palindromes in long reads after whole-genome amplification","volume":"19","author":"Warris","year":"2018","journal-title":"BMC Genom."},{"key":"10.1016\/j.omtm.2022.10.016_bib77","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0070221","article-title":"Automatic nuclei segmentation in H&E stained breast cancer histopathology images","volume":"8","author":"Veta","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2022.10.016_bib78","first-page":"265","article-title":"Medical image computing and computer assisted intervention\u00a0\u2013 MICCAI 2018, 21st international conference, Granada, Spain, september 16-20, 2018, proceedings, Part II","author":"Schmidt","year":"2018"},{"key":"10.1016\/j.omtm.2022.10.016_bib79","doi-asserted-by":"crossref","first-page":"512","DOI":"10.1038\/s41592-018-0011-5","article-title":"CRISPR off-target analysis in genetically engineered rats and mice","volume":"15","author":"Anderson","year":"2018","journal-title":"Nat. Methods"},{"key":"10.1016\/j.omtm.2022.10.016_bib80","doi-asserted-by":"crossref","first-page":"3094","DOI":"10.1093\/bioinformatics\/bty191","article-title":"Minimap2: pairwise alignment for nucleotide sequences","volume":"34","author":"Li","year":"2018","journal-title":"Bioinformatics"},{"key":"10.1016\/j.omtm.2022.10.016_bib81","doi-asserted-by":"crossref","first-page":"2078","DOI":"10.1093\/bioinformatics\/btp352","article-title":"The sequence alignment\/map format and SAMtools","volume":"25","author":"Li","year":"2009","journal-title":"Bioinformatics"},{"key":"10.1016\/j.omtm.2022.10.016_bib82","doi-asserted-by":"crossref","first-page":"841","DOI":"10.1093\/bioinformatics\/btq033","article-title":"BEDTools: a flexible suite of utilities for comparing genomic features","volume":"26","author":"Quinlan","year":"2010","journal-title":"Bioinformatics"},{"key":"10.1016\/j.omtm.2022.10.016_bib83","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0163962","article-title":"SeqKit: a cross-platform and ultrafast toolkit for FASTA\/Q file manipulation","volume":"11","author":"Shen","year":"2016","journal-title":"PLoS One"}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122001589?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122001589?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:31:35Z","timestamp":1762421495000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050122001589"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,12]]},"references-count":83,"alternative-id":["S2329050122001589"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2022.10.016","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2022,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Population-wide gene disruption in the murine lung epithelium via AAV-mediated delivery of CRISPR-Cas9 components","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2022.10.016","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The Author(s).","name":"copyright","label":"Copyright"}]}}