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Updated: Jan 16, 2026

Performing an In Vitro Genome-Wide CRISPR Knockout Screen in Chimeric Antigen Receptor T Cells
Published on: January 31, 2025
In vivo genome-wide CRISPR screens in human T cells to enhance T cell therapy for solid tumors
Qi Liu1,2, Peixin Amy Chen1,2, Esha Urs1,2
1Department of Medicine, University of California, San Francisco, San Francisco, CA, USA.
Abstract:
Large-scale CRISPR screening in human T cells holds significant promise for identifying genetic modifications that can enhance cellular immunotherapy. However, many genetic regulators of T cell performance in solid tumors may not be readily revealed in vitro. In vivo screening in tumor-bearing mice offers greater physiological relevance, but has historically been limited by low intratumoral T cell recovery. Here, we developed a new model system that achieves significantly higher human T cell recovery from tumors, enabling genome-wide in vivo screens with small numbers of mice. Tumor-infiltrating T cells in this model exhibit hallmarks of dysfunction compared to matched splenic T cells, creating an ideal context for screening for genetic modifiers of T cell activity in the tumor microenvironment. Using this platform, we performed two genome-wide CRISPR knockout screens to identify genes regulating T cell intratumoral abundance and effector function (e.g., IFN-γ production). The intratumoral abundance screen uncovered the P2RY8-Gα13 GPCR signaling pathway as a negative regulator of human T cell infiltration into tumors. The effector function screen identified GNAS (Gαs), a central signaling mediator downstream of multiple GPCRs that sense different suppressive ligands, as a key regulator of T cell dysfunction in tumors. Targeted GNAS knockout rendered T cells resistant to multiple suppressive cues and significantly improved therapeutic performance across diverse solid tumor models. Moreover, combinatorial knockout of P2RY8 (trafficking) and GNAS (effector function) further enhanced overall tumor control, demonstrating that genetic modifications targeting distinct T cell phenotypes can be combined to improve therapeutic potency. This flexible and scalable in vivo screening platform can be adapted to diverse tumor models and pooled CRISPR libraries, enabling future discovery of genetic strategies that equip T cell therapies to overcome barriers imposed by solid tumors.
Insights
This study developed an in vivo CRISPR screening model to identify genetic regulators of T cell function in solid tumors. Targeting the P2RY8-Gα13 pathway and GNAS improved T cell infiltration and effector function, enhancing cancer immunotherapy.
Area of Science:
- Immunology
- Cancer Biology
- Genetics
Background:
- In vitro CRISPR screening of human T cells shows promise for immunotherapy but may miss tumor microenvironment regulators.
- In vivo screening offers relevance but is hampered by low T cell recovery in tumors.
Purpose of the Study:
- To develop a novel in vivo model for high-throughput CRISPR screening of human T cells in solid tumors.
- To identify genetic modifiers of T cell intratumoral abundance and effector function.
- To enhance T cell-based cancer immunotherapy.
Main Methods:
- Developed a new mouse model for significantly higher human T cell recovery from tumors.
- Performed genome-wide CRISPR knockout screens to identify genes regulating T cell infiltration and IFN-γ production.
- Utilized targeted knockout of identified genes (P2RY8, GNAS) and combinatorial approaches.
Main Results:
- Identified the P2RY8-Gα13 GPCR signaling pathway as a negative regulator of T cell infiltration.
- Identified GNAS as a key regulator of T cell dysfunction in the tumor microenvironment.
- Demonstrated that GNAS knockout enhances T cell resistance to suppressive cues and improves therapeutic performance.
- Showed that combining P2RY8 and GNAS knockout further improves tumor control.
Conclusions:
- The developed in vivo screening platform enables discovery of genetic strategies to overcome solid tumor barriers for T cell therapies.
- Targeting T cell trafficking (P2RY8) and effector function (GNAS) can be combined for enhanced immunotherapy potency.
- This scalable platform can be adapted for diverse tumor models and CRISPR libraries to advance cellular immunotherapy.

