{"doi":"10.1101/2024.05.03.592383","title":"A temperature-sensitive and interferon-silent Sendai virus vector for CRISPR-Cas9 delivery and gene editing in primary human cells","abstract":"ABSTRACT The transformative potential of gene editing technologies hinges on the development of safe and effective delivery methods. In this study, we developed a temperature-sensitive and interferon-silent Sendai virus (ts SeV) as a novel delivery vector for CRISPR-Cas9 and for efficient gene editing in sensitive human cell types without inducing IFN responses. ts SeV demonstrates unprecedented transduction efficiency in human CD34+ hematopoietic stem and progenitor cells (HSPCs) including transduction of the CD34+/CD38-/CD45RA-/CD90+(Thy1+)/CD49f high stem cell enriched subpopulation. The frequency of CCR5 editing exceeded 90% and bi-allelic CCR5 editing exceeded 70% resulting in significant inhibition of HIV-1 infection in primary human CD14+ monocytes. These results demonstrate the potential of the ts SeV platform as a safe, efficient, and flexible addition to the current gene-editing tool delivery methods, which may help to further expand the possibilities in personalized medicine and the treatment of genetic disorders.","journal":"bioRxiv (Cold Spring Harbor Laboratory)","year":2024,"id":490862,"datarank":0.10397207708399181,"base_score":0.6931471805599453,"endowment":0.6931471805599453,"self_citation_contribution":0.10397207708399181,"citation_network_contribution":0.0,"self_endowment_contribution":0.10397207708399181,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":1,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9522,"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":643187,"name":"Jillian C. Carmichael","orcid":"0000-0001-5270-5916","position":1,"is_corresponding":false},{"id":1316063,"name":"Ruth Watkinson","orcid":"0000-0001-6218-6785","position":2,"is_corresponding":false},{"id":552500,"name":"Shreyas Kowdle","orcid":"0000-0002-7786-3547","position":3,"is_corresponding":false},{"id":1273930,"name":"Rebecca A. Reis","orcid":"0000-0002-6118-1509","position":4,"is_corresponding":false},{"id":1316455,"name":"Kory Hamane","orcid":null,"position":5,"is_corresponding":false},{"id":1316456,"name":"Jason Jang","orcid":null,"position":6,"is_corresponding":false},{"id":520539,"name":"Arnold Park","orcid":"0000-0001-8916-2230","position":7,"is_corresponding":false},{"id":418487,"name":"Olivier Pernet","orcid":"0000-0003-0633-7360","position":8,"is_corresponding":false},{"id":585612,"name":"Wannisa Khamaikawin","orcid":"0000-0001-6766-6841","position":9,"is_corresponding":false},{"id":520540,"name":"Patrick Hong","orcid":"0000-0002-3701-1497","position":10,"is_corresponding":false},{"id":520541,"name":"Patricia A. Thibault","orcid":"0000-0002-7313-0848","position":11,"is_corresponding":false},{"id":553286,"name":"Aditya Gowlikar","orcid":null,"position":12,"is_corresponding":false},{"id":585617,"name":"Dong Sung An","orcid":"0000-0002-7020-9470","position":13,"is_corresponding":false},{"id":110494,"name":"Benhur Lee","orcid":"0000-0003-0760-1709","position":14,"is_corresponding":false},{"id":291520,"name":"Christian S. Stevens","orcid":"0000-0003-2472-6984","position":0,"is_corresponding":true}],"reference_count":74,"raw_metadata":{"citation_network_status":"fetched"},"created_at":"2026-07-19T02:08:37.162700Z","pmid":"38746439","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":[]}