Systematic discovery of CRISPR-boosted CAR T cell immunotherapies

Nature. 2025 Oct;646(8086):963-972. doi: 10.1038/s41586-025-09507-9. Epub 2025 Sep 24.

Abstract

Chimeric antigen receptor (CAR) T cell therapy has shown remarkable success in treating blood cancers, but CAR T cell dysfunction remains a common cause of treatment failure1. Here we present CELLFIE, a CRISPR screening platform for enhancing CAR T cells across multiple clinical objectives. We performed genome-wide screens in human primary CAR T cells, with readouts capturing key aspects of T cell biology, including proliferation, target cell recognition, activation, apoptosis and fratricide, and exhaustion. Screening hits were prioritized using a new in vivo CROP-seq2 method in a xenograft model of human leukaemia, establishing several gene knockouts that boost CAR T cell efficacy. Most notably, we discovered that RHOG knockout is a potent and unexpected CAR T cell enhancer, both individually and together with FAS knockout, which was validated across multiple in vivo models, CAR designs and sample donors, and in patient-derived cells. Demonstrating the versatility of the CELLFIE platform, we also conducted combinatorial CRISPR screens to identify synergistic gene pairs and saturation base-editing screens to characterize RHOG variants. In summary, we discovered, validated and biologically characterized CRISPR-boosted CAR T cells that outperform standard CAR T cells in widely used benchmarks, establishing a foundational resource for optimizing cell-based immunotherapies.

MeSH terms

  • Animals
  • CRISPR-Cas Systems* / genetics
  • Clustered Regularly Interspaced Short Palindromic Repeats* / genetics
  • Female
  • Gene Editing
  • Gene Knockout Techniques
  • Humans
  • Immunotherapy* / methods
  • Immunotherapy, Adoptive* / methods
  • Leukemia / genetics
  • Leukemia / immunology
  • Leukemia / therapy
  • Male
  • Mice
  • Receptors, Chimeric Antigen* / genetics
  • Receptors, Chimeric Antigen* / immunology
  • Receptors, Chimeric Antigen* / metabolism
  • T-Lymphocytes* / cytology
  • T-Lymphocytes* / immunology
  • T-Lymphocytes* / metabolism
  • Xenograft Model Antitumor Assays
  • rho GTP-Binding Proteins / deficiency
  • rho GTP-Binding Proteins / genetics

Substances

  • Receptors, Chimeric Antigen
  • rho GTP-Binding Proteins