Amelioration of colitis by genetically engineered murine regulatory T cells redirected by antigen-specific chimeric receptor

Gastroenterology. 2009 May;136(5):1721-31. doi: 10.1053/j.gastro.2009.01.049. Epub 2009 Jan 27.

Abstract

Background & aims: The therapeutic application of regulatory T cells (Tregs) for the treatment of inflammatory diseases is limited by the scarcity of antigen-specific Tregs. A preferred approach to endow effector T cells (Teff) with a desired specificity uses chimeric immune receptors with antibody-type specificity. Accordingly, employing such chimeric immune receptors to redirect Tregs to sites of inflammation may be a useful therapeutic approach to alleviate a broad scope of diseases in which an uncontrolled inflammatory response plays a major role.

Methods: To enable application of the approach in clinical setting, which requires the genetic modification of the patient's own Tregs, we describe here a novel protocol that allows the efficient retroviral transduction and 2,4,6-trinitrophenol-specific expansion of murine naturally occurring regulatory T cells (nTregs), with a 2,4,6-trinitrophenol-specific tripartite chimeric receptor.

Results: Transduced Tregs maintained their Foxp3 level, could undergo repeated expansion upon ex vivo encounter with their cognate antigen in a major histocompatibility complex-independent, costimulation-independent, and contact-dependent manner and specifically suppressed Teff cells. Adoptive transfer of small numbers of the transduced nTregs was associated with antigen-specific, dose-dependent amelioration of trinitrobenzenesulphonic acid colitis.

Conclusions: This study demonstrates that nTregs can be efficiently transduced to express functional, antigen-specific chimeric receptors that enable the specific suppression of effector T cells both in vitro and in vivo. This approach may enable future cell-based therapeutic application in inflammatory bowel disease, as well as other inflammatory disorders.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adoptive Transfer
  • Animals
  • Antibody Specificity*
  • Colitis / immunology
  • Colitis / therapy*
  • Female
  • Gene Products, env / immunology
  • Gene Products, gag / immunology
  • Gene Products, pol / immunology
  • Gene Transfer Techniques
  • Genetic Engineering*
  • Genetic Vectors
  • Mice
  • Mice, Inbred BALB C
  • Mice, Inbred C57BL
  • Mice, Transgenic
  • Receptors, Antigen / immunology*
  • Recombinant Fusion Proteins / immunology
  • Retroviridae / genetics
  • T-Lymphocytes, Regulatory / immunology*

Substances

  • Gene Products, env
  • Gene Products, gag
  • Gene Products, pol
  • Receptors, Antigen
  • Recombinant Fusion Proteins