{"doi":"10.1089/hum.2024.035","title":"AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys","abstract":"Genome editing has the potential to treat genetic diseases in a variety of tissues, including the lung. We have previously developed and validated a dual adeno-associated virus (AAV) CRISPR platform that supports effective editing in the airways of mice. To validate this delivery vehicle in a large animal model, we have shown that intratracheal instillation of CRISPR/Cas9 in AAV5 can edit a housekeeping gene or a disease-related gene in the lungs of young rhesus monkeys. We observed up to 8% editing of angiotensin-converting enzyme 2 (ACE2) in lung lobes after single-dose administration. Single-nuclear RNA sequencing revealed that AAV5 transduces multiple cell types in the caudal lung lobes, including alveolar cells, macrophages, fibroblasts, endothelial cells, and B cells. These results demonstrate that AAV5 is efficient in the delivery of CRISPR/Cas9 in the lung lobes of young rhesus monkeys.","journal":"Human Gene Therapy","year":2024,"id":444694,"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":7,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9449,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2024-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":558593,"name":"Andrew W. Navia","orcid":"0000-0002-5429-8012","position":1,"is_corresponding":false},{"id":1017944,"name":"Michelle L. Ramseier","orcid":"0000-0002-9201-7656","position":2,"is_corresponding":false},{"id":1075278,"name":"Xuntao Zhou","orcid":null,"position":3,"is_corresponding":false},{"id":1260635,"name":"Michele Martinez","orcid":null,"position":4,"is_corresponding":false},{"id":1260160,"name":"Charles K. Lee","orcid":"0000-0002-6562-4733","position":5,"is_corresponding":false},{"id":1260161,"name":"Chen Zhou","orcid":"0000-0001-6782-2277","position":6,"is_corresponding":false},{"id":1260636,"name":"Joae Wu","orcid":null,"position":7,"is_corresponding":false},{"id":344875,"name":"Jun Xie","orcid":"0000-0001-9565-1567","position":8,"is_corresponding":false},{"id":323183,"name":"Qin Su","orcid":"0000-0001-9437-6292","position":9,"is_corresponding":false},{"id":226489,"name":"Dan Wang","orcid":"0000-0001-9079-2360","position":10,"is_corresponding":false},{"id":460610,"name":"Terence R. Flotte","orcid":"0000-0002-8255-0588","position":11,"is_corresponding":false},{"id":60783,"name":"Daniel G. Anderson","orcid":"0000-0001-5629-4798","position":12,"is_corresponding":false},{"id":306070,"name":"Alice F. Tarantal","orcid":null,"position":13,"is_corresponding":false},{"id":1413,"name":"Alex K. Shalek","orcid":"0000-0001-5670-8778","position":14,"is_corresponding":false},{"id":226490,"name":"Guangping Gao","orcid":"0000-0003-0097-9012","position":15,"is_corresponding":false},{"id":265835,"name":"Wen Xue","orcid":"0000-0002-9797-8042","position":16,"is_corresponding":false},{"id":362071,"name":"Shun‐Qing Liang","orcid":"0000-0003-4664-6029","position":0,"is_corresponding":true}],"reference_count":37,"raw_metadata":null,"created_at":"2026-07-19T02:01:37.886033Z","pmid":"38767512","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":[]}