{"doi":"10.1371/journal.pone.0295009","title":"Prime editing-mediated correction of the CFTR W1282X mutation in iPSCs and derived airway epithelial cells","abstract":"A major unmet need in the cystic fibrosis (CF) therapeutic landscape is the lack of effective treatments for nonsense CFTR mutations, which affect approximately 10% of CF patients. Correction of nonsense CFTR 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 CFTR mutations. We generated iPSCs from a CF patient homozygous for the CFTR 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 CFTR mutations such as W1282X.","journal":"PLoS ONE","year":2023,"id":336698,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":19,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9512,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":544303,"name":"Zhong Liu","orcid":"0000-0002-5132-7463","position":1,"is_corresponding":false},{"id":902085,"name":"Justin D. Anderson","orcid":"0000-0002-8894-3876","position":2,"is_corresponding":false},{"id":902086,"name":"Zhongyu Liu","orcid":"0000-0001-7385-5681","position":3,"is_corresponding":false},{"id":283904,"name":"Liping Tang","orcid":"0000-0002-3393-7073","position":4,"is_corresponding":false},{"id":662104,"name":"Li Yao","orcid":"0000-0002-7785-5563","position":5,"is_corresponding":false},{"id":426129,"name":"Ning Peng","orcid":"0000-0003-4213-1159","position":6,"is_corresponding":false},{"id":603476,"name":"Jianguo Chen","orcid":"0000-0002-7805-3589","position":7,"is_corresponding":false},{"id":1067599,"name":"Xueming Liu","orcid":"0000-0003-0676-9745","position":8,"is_corresponding":false},{"id":429080,"name":"Lianwu Fu","orcid":null,"position":9,"is_corresponding":false},{"id":695901,"name":"Tim M. Townes","orcid":null,"position":10,"is_corresponding":false},{"id":263850,"name":"Steven M. Rowe","orcid":"0000-0001-9045-0133","position":11,"is_corresponding":false},{"id":631078,"name":"David M. Bedwell","orcid":"0000-0002-6605-818X","position":12,"is_corresponding":false},{"id":317013,"name":"Jennifer S. Guimbellot","orcid":"0000-0002-0514-8536","position":13,"is_corresponding":false},{"id":1067600,"name":"Rui Zhao","orcid":"0009-0009-3031-8435","position":14,"is_corresponding":false},{"id":1067598,"name":"Chao Li","orcid":"0000-0002-2769-7616","position":0,"is_corresponding":true}],"reference_count":63,"raw_metadata":null,"created_at":"2026-07-19T01:10:21.947540Z","pmid":"38019847","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}