Orthotopic T-Cell Receptor Replacement-An "Enabler" for TCR-Based Therapies

Cells. 2020 Jun 1;9(6):1367. doi: 10.3390/cells9061367.

Abstract

Natural adaptive immunity co-evolved with pathogens over millions of years, and adoptive transfer of non-engineered T cells to fight infections or cancer so far exhibits an exceptionally safe and functional therapeutic profile in clinical trials. However, the personalized nature of therapies using virus-specific T cells, donor lymphocyte infusion, or tumor-infiltrating lymphocytes makes implementation in routine clinical care difficult. In principle, genetic engineering can be used to make T-cell therapies more broadly applicable, but so far it significantly alters the physiology of cells. We recently demonstrated that orthotopic T-cell receptor (TCR) replacement (OTR) by clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR-associated protein 9 (Cas9) can be used to generate engineered T cells with preservation of near-physiological function. In this review, we present the current status of OTR technology development and discuss its potential for TCR-based therapies. By providing the means to combine the therapeutic efficacy and safety profile of physiological T cells with the versatility of cell engineering, OTR can serve as an "enabler" for TCR-based therapies.

Keywords: CRISPR/Cas9; OTR; T-cell therapy; TCR engineering; adoptive cell therapy; gene editing; gene therapy; immunotherapy; orthotopic TCR replacement.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antigens / metabolism
  • Gene Editing
  • Genetic Therapy
  • Humans
  • Immunotherapy*
  • Receptors, Antigen, T-Cell / metabolism*
  • Translational Research, Biomedical

Substances

  • Antigens
  • Receptors, Antigen, T-Cell