{"id":"doi:10.1038/s41408-021-00508-1","kind":"article","title":"AAV-mediated in vivo CAR gene therapy for targeting human T-cell leukemia","authors":["Nawaz W","Huang B","Xu S","Li Y","Zhu L","Yiqiao H","Wu Z","Wu X"],"author_count":8,"year":2021,"published":"2021-06-23","venue":"Blood cancer journal","cited_by":134,"doi":"10.1038/s41408-021-00508-1","arxiv_id":null,"nct_id":null,"source_url":"https://doi.org/10.1038/s41408-021-00508-1","archive_url":"https://space-kitty.com/papers/doi:10.1038/s41408-021-00508-1","updated":null,"pmid":"34162832","pmcid":"PMC8222347","openalex_id":null,"pdf_url":"https://europepmc.org/articles/PMC8222347/pdf","abstract":"Chimeric antigen receptor (CAR) T-cell therapy is the most active field in immuno-oncology and brings substantial benefit to patients with B cell malignancies. However, the complex procedure for CAR T-cell generation hampers its widespread applications. Here, we describe a novel approach in which human CAR T cells can be generated within the host upon injecting an Adeno-associated virus (AAV) vector carrying the CAR gene, which we call AAV delivering CAR gene therapy (ACG). Upon single infusion into a humanized NOD.Cg-Prkd cscid Il2rg em26 /Nju tumor mouse model of human T-cell leukemia, AAV generates sufficient numbers of potent in vivo CAR cells, resulting in tumor regression; these in vivo-generated CAR cells produce antitumor immunological characteristics. This instantaneous generation of in vivo CAR T cells may bypass the need for patient lymphodepletion, as well as the β processes of traditional CAR T-cell production, which may make CAR therapy simpler and less expensive. It may allow the development of intricate, individualized treatments in the form of on-demand and diverse therapies.","abstract_license":"CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)","license":"CC-BY","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":[],"details":null,"provenance":"europepmc","attribution":"Abstract of \"AAV-mediated in vivo CAR gene therapy for targeting human T-cell leukemia\" by Nawaz W, Huang B, Xu S et al., https://doi.org/10.1038/s41408-021-00508-1, used under its Creative Commons Attribution license (CC-BY); markup removed, otherwise unmodified.","topics":[{"slug":"gene-cell-therapy","name":"Gene & Cell Therapy","domain_slug":"medicine"}],"cited_in_dossiers":[]}