Simple conditioning with monospecific CD4+CD25+ regulatory T cells for bone marrow engraftment and tolerance to multiple gene products

Blood. 2006 Sep 15;108(6):1841-8. doi: 10.1182/blood-2006-02-011981. Epub 2006 Jun 1.

Abstract

A major impediment to gene replacement therapy is immune elimination of genetically modified cells. In principle, this can be dealt with by inducing a strong, specific, and enduring tolerance through engraftment of transgene-modified autologous bone marrow (BM). Because usual myeloablation and/or immunosuppression are risk factors in most pathologies, we assessed the potential of monospecific CD4(+)CD25(+) regulatory T cells (Tregs) to engraft minor-mismatched BM without preconditioning. We found that as few as 5 x 10(4) Tregs directed to the male DBY protein promote the engraftment of foreign male BM into sex-mismatched female hosts, establishing sustained chimerism in all hematopoeitic compartments. We achieved concomitantly strong tolerance to all foreign antigens expressed in the BM, likely occurring through induction of anergy and/or deletion of antidonor T cells. Chimerism was obtained in thymectomized mice too, underlining the major role of peripheral tolerance mechanisms in our system. This allowed us to engraft gene-modified tissues while preserving full immunocompetence to third-party antigens. Our results demonstrate that very few donor-specific Tregs are effective as the sole conditioning to induce mixed molecular chimerism and long-term tolerance to multiple foreign antigens.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Base Sequence
  • Bone Marrow Transplantation / immunology*
  • Chimera / immunology
  • DEAD-box RNA Helicases
  • Female
  • Forkhead Transcription Factors / genetics
  • Green Fluorescent Proteins / genetics
  • Green Fluorescent Proteins / immunology
  • Immune Tolerance
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Knockout
  • Mice, Transgenic
  • Minor Histocompatibility Antigens
  • Proteins / genetics
  • Proteins / immunology
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Skin Transplantation / immunology
  • T-Lymphocytes, Regulatory / immunology*
  • Tissue Donors
  • Transplantation Conditioning / methods*
  • Transplantation, Homologous

Substances

  • Forkhead Transcription Factors
  • Foxp3 protein, mouse
  • Minor Histocompatibility Antigens
  • Proteins
  • RNA, Messenger
  • enhanced green fluorescent protein
  • Green Fluorescent Proteins
  • Ddx3y protein, mouse
  • DEAD-box RNA Helicases