We found a match
Your institution may have rights to this item. Sign in to continue.
- Title
AAV-mediated in vivo CAR gene therapy for targeting human T-cell leukemia.
- Authors
Nawaz, Waqas; Huang, Bilian; Xu, Shijie; Li, Yanlei; Zhu, Linjing; Yiqiao, Hu; Wu, Zhiwei; Wu, Xilin
- 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-Prkdcscid Il2rgem26/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.
- Subjects
GENE therapy; LEUKEMIA; CHIMERIC antigen receptors; ADENOVIRUSES; B cell lymphoma
- Publication
Blood Cancer Journal, 2021, Vol 11, Issue 6, p1
- ISSN
2044-5385
- Publication type
Article
- DOI
10.1038/s41408-021-00508-1