CAR Treg cells mediate linked suppression and infectious tolerance in islet transplantation in mice

Sci Transl Med. 2025 Aug 20;17(812):eadp6519. doi: 10.1126/scitranslmed.adp6519. Epub 2025 Aug 20.

Abstract

Regulatory T cells (Treg cells) have potential as a cell-based therapy to prevent or treat transplant rejection and autoimmunity. Using a human leukocyte antigen (HLA)-A2-specific chimeric antigen receptor (A2-CAR), we previously showed that adoptive transfer of A2-CAR Treg cells can limit anti-HLA-A2 alloimmunity. However, it was unknown whether A2-CAR Treg cells could also limit immunity to autoantigens. Using a model of HLA-A2+ islet transplantation into immunodeficient nonobese diabetic mice, we investigated whether A2-CAR Treg cells could control hyperglycemia induced by diabetogenic BDC2.5 effector T cells. In mice transplanted with HLA-A2+ islets, A2-CAR Treg cells reduced BDC2.5 T cell engraftment, proliferation, and cytokine production and protected mice from diabetes. Islet tolerance was systemic, including protection of the HLA-A2negative endogenous pancreas. Treated mice remained euglycemic even after removal of the HLA-A2+ islet graft and A2-CAR Treg cells. Thus, A2-CAR Treg cells can induce linked suppression and long-lasting tolerance to a distinct autoimmune antigen. Tolerance to the autoantigen does not require A2-CAR Treg persistence, indicating the presence of infectious tolerance. Overall, these data demonstrate that A2-CAR Treg cells have potential therapeutic use to simultaneously control both allo- and autoimmunity in islet transplantation.

MeSH terms

  • Animals
  • Autoantigens / immunology
  • Diabetes Mellitus, Experimental / immunology
  • Diabetes Mellitus, Experimental / therapy
  • HLA-A2 Antigen / immunology
  • HLA-A2 Antigen / metabolism
  • Humans
  • Immune Tolerance* / immunology
  • Islets of Langerhans Transplantation* / immunology
  • Mice
  • Mice, Inbred NOD
  • Receptors, Chimeric Antigen* / immunology
  • Receptors, Chimeric Antigen* / metabolism
  • T-Lymphocytes, Regulatory* / immunology

Substances

  • Receptors, Chimeric Antigen
  • HLA-A2 Antigen
  • Autoantigens