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Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection.

作者信息

Eyquem Justin, Mansilla-Soto Jorge, Giavridis Theodoros, van der Stegen Sjoukje J C, Hamieh Mohamad, Cunanan Kristen M, Odak Ashlesha, Gönen Mithat, Sadelain Michel

机构信息

Center for Cell Engineering and Immunology Program, Sloan Kettering Institute, New York, New York 10065, USA.

Department of Epidemiology and Biostatistics, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA.

出版信息

Nature. 2017 Mar 2;543(7643):113-117. doi: 10.1038/nature21405. Epub 2017 Feb 22.


DOI:10.1038/nature21405
PMID:28225754
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5558614/
Abstract

Chimeric antigen receptors (CARs) are synthetic receptors that redirect and reprogram T cells to mediate tumour rejection. The most successful CARs used to date are those targeting CD19 (ref. 2), which offer the prospect of complete remission in patients with chemorefractory or relapsed B-cell malignancies. CARs are typically transduced into the T cells of a patient using γ-retroviral vectors or other randomly integrating vectors, which may result in clonal expansion, oncogenic transformation, variegated transgene expression and transcriptional silencing. Recent advances in genome editing enable efficient sequence-specific interventions in human cells, including targeted gene delivery to the CCR5 and AAVS1 loci. Here we show that directing a CD19-specific CAR to the T-cell receptor α constant (TRAC) locus not only results in uniform CAR expression in human peripheral blood T cells, but also enhances T-cell potency, with edited cells vastly outperforming conventionally generated CAR T cells in a mouse model of acute lymphoblastic leukaemia. We further demonstrate that targeting the CAR to the TRAC locus averts tonic CAR signalling and establishes effective internalization and re-expression of the CAR following single or repeated exposure to antigen, delaying effector T-cell differentiation and exhaustion. These findings uncover facets of CAR immunobiology and underscore the potential of CRISPR/Cas9 genome editing to advance immunotherapies.

摘要

相似文献

[1]
Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection.

Nature. 2017-3-2

[2]
Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells.

Mol Ther. 2017-4-5

[3]
CRISPR/Cas9-Engineered Universal CD19/CD22 Dual-Targeted CAR-T Cell Therapy for Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia.

Clin Cancer Res. 2021-5-15

[4]
Long Terminal Repeat CRISPR-CAR-Coupled "Universal" T Cells Mediate Potent Anti-leukemic Effects.

Mol Ther. 2018-3-6

[5]
CRISPR/Cas9-based genome editing in the era of CAR T cell immunotherapy.

Hum Vaccin Immunother. 2019-4-2

[6]
CD19 CAR T Cells.

Cell. 2017-12-14

[7]
High Cytotoxic Efficiency of Lentivirally and Alpharetrovirally Engineered CD19-Specific Chimeric Antigen Receptor Natural Killer Cells Against Acute Lymphoblastic Leukemia.

Front Immunol. 2020-1-24

[8]
CAR T Cell Therapy in Acute Lymphoblastic Leukemia and Potential for Chronic Lymphocytic Leukemia.

Curr Treat Options Oncol. 2016-6

[9]
CD19 CAR-targeted T cells induce long-term remission and B Cell Aplasia in an immunocompetent mouse model of B cell acute lymphoblastic leukemia.

PLoS One. 2013-4-9

[10]
CRISPR/Cas9-mediated PD-1 disruption enhances anti-tumor efficacy of human chimeric antigen receptor T cells.

Sci Rep. 2017-4-7

引用本文的文献

[1]
Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases.

Mol Ther Nucleic Acids. 2025-8-5

[2]
Recent advances in universal chimeric antigen receptor T cell therapy.

J Hematol Oncol. 2025-8-29

[3]
CRISPR tools for T cells: targeting the genome, epigenome, and transcriptome.

Trends Cancer. 2025-8-28

[4]
Directed evolution-based discovery of ligands for in vivo restimulation of chimeric antigen receptor T cells.

Nat Biomed Eng. 2025-8-25

[5]
ONE-STEP tagging: a versatile method for rapid site-specific integration by simultaneous reagent delivery.

Nucleic Acids Res. 2025-8-11

[6]
production of CAR T cell: Opportunities and challenges.

Genes Dis. 2025-3-25

[7]
Exploiting TCR Repertoire Analysis to Select Therapeutic TCRs for Cancer Immunotherapy.

Cells. 2025-8-7

[8]
A Singular Base Editing Platform for Polyfunctional Multiplex Engineering of Immune Cells.

bioRxiv. 2025-7-16

[9]
Miniaturized scalable arrayed CRISPR screening in primary cells enables discovery at the single donor resolution.

Sci Rep. 2025-8-11

[10]
Tracing the development of CAR-T cell design: from concept to next-generation platforms.

Front Immunol. 2025-7-17

本文引用的文献

[1]
Affinity and dose of TCR engagement yield proportional enhancer and gene activity in CD4+ T cells.

Elife. 2016-7-4

[2]
Clinical manufacturing of CAR T cells: foundation of a promising therapy.

Mol Ther Oncolytics. 2016-6-15

[3]
Defining and improving the genome-wide specificities of CRISPR-Cas9 nucleases.

Nat Rev Genet. 2016-5

[4]
Targeted gene editing restores regulated CD40L function in X-linked hyper-IgM syndrome.

Blood. 2016-2-22

[5]
Biology and Applications of CRISPR Systems: Harnessing Nature's Toolbox for Genome Engineering.

Cell. 2016-1-14

[6]
Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.

Nucleic Acids Res. 2016-2-18

[7]
Structural Design of Engineered Costimulation Determines Tumor Rejection Kinetics and Persistence of CAR T Cells.

Cancer Cell. 2015-10-12

[8]
Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template.

Sci Transl Med. 2015-9-30

[9]
Chimeric antigen receptor-modified T cells derived from defined CD8+ and CD4+ subsets confer superior antitumor reactivity in vivo.

Leukemia. 2016-2

[10]
CAR therapy: the CD19 paradigm.

J Clin Invest. 2015-9

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