Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL.

ABSTRACT: Recently, chimeric antigen receptor (CAR)-T cell therapy has shown great promise in treating haematological malignancies^1-7. However, CAR-T cell therapy currently has several limitations^8-12. Here we successfully developed a two-in-one approach to generate non-viral, gene-specific targeted CAR-T cells through CRISPR-Cas9. Using the optimized protocol, we demonstrated feasibility in a preclinical study by inserting an anti-CD19 CAR cassette into the AAVS1 safe-harbour locus. Furthermore, an innovative type of anti-CD19 CAR-T cell with PD1 integration was developed and showed superior ability to eradicate tumour cells in xenograft models. In adoptive therapy for relapsed/refractory aggressive B cell non-Hodgkin lymphoma (ClinicalTrials.gov, NCT04213469 ), we observed a

SUBMITTER: Zhang J 

PROVIDER: S-EPMC9452296 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature