# AAV-mediated in vivo CAR gene therapy for targeting human T-cell leukemia

Article · Nawaz W, Huang B, Xu S, Li Y, Zhu L, Yiqiao H, Wu Z, Wu X

- Archive id: doi:10.1038/s41408-021-00508-1
- Published: 2021-06-23
- Venue: Blood cancer journal
- DOI: https://doi.org/10.1038/s41408-021-00508-1
- PMID: 34162832
- PMCID: PMC8222347
- Citations: 134
- Source URL: https://doi.org/10.1038/s41408-021-00508-1
- PDF: https://europepmc.org/articles/PMC8222347/pdf
- Provenance: europepmc

## 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 reproduced under CC-BY (article license as recorded by Europe PMC; abstract reproduced unmodified apart from markup removal)._

Topics: [Gene & Cell Therapy](https://space-kitty.com/topics/gene-cell-therapy)

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