{"doi":"10.31219/osf.io/d26uf","title":"In vivo gene delivery to immune cells","abstract":"Immune cell therapies are an emerging class of living drugs that have the potential to cure patients with serious or life-threatening diseases as exemplified by the landmark approval of chimeric antigen receptor (CAR) T cell therapy to treat patients with certain types of hematological malignancies. This category of medicine relies on the delivery of therapeutic transgenes that can enhance, modulate or restore the function of target immune cells such as transgenes that encode for tumor-targeting receptors or for functional proteins to replace dysfunctional ones. However, many cellular immunotherapies are autologous therapies that are limited by their high manufacturing costs, typical vein-to-vein time of 3 to 4 weeks, and severe immune-related adverse effects that can be life-threatening. To address these issues, gene delivery vehicles such as lipid nanoparticles, polymeric nanoparticles, viruses, and virus-like particles, are being developed to target specific immune cell subsets in vivo to bypass the limitations of ex vivo manufacturing, modulate therapeutic responses in situ and improve safety by reducing on- and off-target toxicity. The success of in vivo gene delivery to immune cells – which is being tested at the preclinical and clinical stages of development for the treatment of cancer, infectious diseases, and autoimmunity – is paramount for the democratization of cellular immunotherapies.","journal":"OSF Preprints (OSF Preprints)","year":2023,"id":403605,"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":0,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9518,"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":1181001,"name":"Ching Shen Chan","orcid":"0000-0001-7794-493X","position":1,"is_corresponding":false},{"id":853800,"name":"Xinyi Yao","orcid":null,"position":2,"is_corresponding":false},{"id":374174,"name":"Fang‐Yi Su","orcid":"0000-0003-2852-161X","position":3,"is_corresponding":false},{"id":497869,"name":"Gabriel A. Kwong","orcid":"0000-0002-6255-6755","position":4,"is_corresponding":false},{"id":1181332,"name":"Jamison Charles Siebart","orcid":null,"position":0,"is_corresponding":true}],"reference_count":57,"raw_metadata":null,"created_at":"2026-07-19T01:20:36.280647Z","pmid":null,"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":[]}