Multiplexed activation of endogenous genes by CRISPRa elicits potent antitumor immunity

Nat Immunol. 2019 Nov;20(11):1494-1505. doi: 10.1038/s41590-019-0500-4. Epub 2019 Oct 14.

Abstract

Immunotherapy has transformed cancer treatment. However, current immunotherapy modalities face various limitations. In the present study, we developed multiplexed activation of endogenous genes as an immunotherapy (MAEGI), a new form of immunotherapy that elicits antitumor immunity through multiplexed activation of endogenous genes in tumors. We leveraged CRISPR activation (CRISPRa) to directly augment the in situ expression of endogenous genes, and thereby the presentation of tumor antigens, leading to dramatic antitumor immune responses. Deploying this as a cell-based vaccination strategy showed efficacy in both prophylactic and therapeutic settings. Intratumoral adeno-associated virus delivery of CRISPRa libraries elicited strong antitumor immunity across multiple cancer types. Precision targeting of mutated gene sets eradicated a large fraction of established tumors at both local and distant sites. This treatment modality led to alterations in the tumor microenvironment, marked by enhanced T cell infiltration and antitumor immune signatures. Multiplexed endogenous gene activation is a versatile and highly scalable strategy to elicit potent immune responses against cancer, distinct from all existing cancer therapies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Antigen Presentation / genetics
  • Antigens, Neoplasm / immunology
  • Cancer Vaccines / administration & dosage
  • Cancer Vaccines / immunology
  • Cell Line, Tumor
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Coculture Techniques
  • Combined Modality Therapy / methods
  • Dependovirus / genetics
  • Disease Models, Animal
  • Female
  • Gene Expression Regulation, Neoplastic / immunology*
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage
  • Genetic Vectors / genetics
  • HEK293 Cells
  • Humans
  • Immunotherapy / methods*
  • Injections, Intralesional
  • Lymphocytes, Tumor-Infiltrating / immunology
  • Male
  • Mice
  • Neoplasms / drug therapy*
  • Neoplasms / genetics
  • Neoplasms / immunology
  • T-Lymphocytes, Cytotoxic / immunology
  • Tumor Microenvironment / genetics
  • Tumor Microenvironment / immunology

Substances

  • Antigens, Neoplasm
  • Cancer Vaccines