Conversion of pathogenic T cells into functionally stabilized Treg cells for antigen-specific immunosuppression in pemphigus vulgaris

Sci Transl Med. 2025 Oct 22;17(821):eadq9913. doi: 10.1126/scitranslmed.adq9913. Epub 2025 Oct 22.

Abstract

Antigen-specific immunotherapy represents one candidate strategy for treating autoimmune diseases such as pemphigus vulgaris, a skin autoimmune disorder mediated by anti-desmoglein 3 (Dsg3) autoantibodies. We developed a therapeutic strategy by which Dsg3-specific pathogenic autoreactive CD4+ T cells were converted in vitro into functionally stable Foxp3+ regulatory T (Treg) cells, designated stable and functional induced Treg (S/F-iTreg) cells. The conversion was achieved by pharmacological induction of Foxp3 and costimulation-dependent installation of Treg cell-specific epigenetic changes. In an animal model of pemphigus vulgaris, the Dsg3-specific S/F-iTreg cells expanded specifically in the skin-draining lymph nodes through recognition of endogenous Dsg3. They selectively inhibited Dsg3-specific T follicular helper cell and B cell proliferation and, consequently, anti-Dsg3 autoantibody formation, without affecting the total B cell population, thereby mitigating disease progression without inducing systemic immunosuppression. Human S/F-iTreg cells with similar functions could also be efficiently generated from peripheral blood T cells of patients with pemphigus vulgaris. This study demonstrates that pathogenic autoreactive T cells can be converted into disease-specific Treg cells retaining antigen specificity, enabling antigen- and disease-specific treatment of autoimmune disease.

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Animals
  • Autoantibodies / immunology
  • Autoantibodies / metabolism
  • B-Lymphocytes / immunology
  • CD4-Positive T-Lymphocytes* / drug effects
  • CD4-Positive T-Lymphocytes* / immunology
  • CD4-Positive T-Lymphocytes* / metabolism
  • Case-Control Studies
  • Cell Proliferation
  • Desmoglein 3 / genetics
  • Desmoglein 3 / immunology
  • Disease Models, Animal
  • Female
  • Forkhead Transcription Factors / agonists
  • Forkhead Transcription Factors / metabolism
  • Humans
  • Immunosuppression Therapy* / methods
  • Lymph Nodes / cytology
  • Lymph Nodes / immunology
  • Lymph Nodes / pathology
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Middle Aged
  • Pemphigus* / blood
  • Pemphigus* / immunology
  • Pemphigus* / pathology
  • Pemphigus* / therapy
  • Primary Cell Culture
  • Skin / immunology
  • Skin / pathology
  • T-Lymphocytes, Regulatory* / immunology
  • T-Lymphocytes, Regulatory* / metabolism
  • Young Adult

Substances

  • Autoantibodies
  • Desmoglein 3
  • Forkhead Transcription Factors