{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,25]],"date-time":"2026-10-25T20:25:02Z","timestamp":1792959902772,"version":"4.5.0"},"reference-count":61,"publisher":"Public Library of Science (PLoS)","issue":"11","license":[{"start":{"date-parts":[[2023,11,29]],"date-time":"2023-11-29T00:00:00Z","timestamp":1701216000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","award":["ZHAO19G0"],"award-info":[{"award-number":["ZHAO19G0"]}],"id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000179","name":"Office of the Director","doi-asserted-by":"publisher","award":["R01OD026594"],"award-info":[{"award-number":["R01OD026594"]}],"id":[{"id":"10.13039\/100000179","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008333","name":"University of Alabama at Birmingham","doi-asserted-by":"publisher","award":["UAB Cystic Fibrosis Research Center Pilot Grant (ROWE15R0)"],"award-info":[{"award-number":["UAB Cystic Fibrosis Research Center Pilot Grant (ROWE15R0)"]}],"id":[{"id":"10.13039\/100008333","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"abstract":"<jats:p>A major unmet need in the cystic fibrosis (CF) therapeutic landscape is the lack of effective treatments for nonsense <jats:italic>CFTR<\/jats:italic> mutations, which affect approximately 10% of CF patients. Correction of nonsense <jats:italic>CFTR<\/jats:italic> mutations via genomic editing represents a promising therapeutic approach. In this study, we tested whether prime editing, a novel CRISPR-based genomic editing method, can be a potential therapeutic modality to correct nonsense <jats:italic>CFTR<\/jats:italic> mutations. We generated iPSCs from a CF patient homozygous for the <jats:italic>CFTR<\/jats:italic> W1282X mutation. We demonstrated that prime editing corrected one mutant allele in iPSCs, which effectively restored CFTR function in iPSC-derived airway epithelial cells and organoids. We further demonstrated that prime editing may directly repair mutations in iPSC-derived airway epithelial cells when the prime editing machinery is efficiently delivered by helper-dependent adenovirus (HDAd). Together, our data demonstrated that prime editing may potentially be applied to correct <jats:italic>CFTR<\/jats:italic> mutations such as W1282X.<\/jats:p>","DOI":"10.1371\/journal.pone.0295009","type":"journal-article","created":{"date-parts":[[2023,11,29]],"date-time":"2023-11-29T18:33:12Z","timestamp":1701282792000},"page":"e0295009","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":18,"title":["Prime editing-mediated correction of the CFTR W1282X mutation in iPSCs and derived airway epithelial cells"],"prefix":"10.1371","volume":"18","author":[{"given":"Chao","family":"Li","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhong","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8894-3876","authenticated-orcid":true,"given":"Justin","family":"Anderson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhongyu","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liping","family":"Tang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7785-5563","authenticated-orcid":true,"given":"Yao","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ning","family":"Peng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianguo","family":"Chen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xueming","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lianwu","family":"Fu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tim 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