Tweaking the immune system: Gene therapy-assisted autologous haematopoietic stem cell transplantation as a treatment for autoimmune disease

References

• Janeway CA, Jr., Medzhitov R. Innate immune recognition. Annu Rev Immunol 2002; 20: 197–216
   PubMed Web of Science ®Google Scholar
• Diamond B. Autoimmunity. Immunol Rev 2005; 204: 5–8
   PubMed Web of Science ®Google Scholar
• Hogquist KA, Baldwin TA, Jameson SC. Central tolerance: Learning self-control in the thymus. Nat Rev Immunol 2005; 5(10)772–782
   PubMed Web of Science ®Google Scholar
• von Boehmer H. Mechanisms of suppression by suppressor T cells. Nat Immunol 2005; 6(4)338–344
   PubMed Web of Science ®Google Scholar
• Miyara M, Sakaguchi S. Natural regulatory T cells: Mechanisms of suppression. Trends Mol Med 2007; 13(3)108–116
   PubMed Web of Science ®Google Scholar
• Yamaguchi T, Hirota K, Nagahama K, et al. Control of immune responses by antigen-specific regulatory T cells expressing the folate receptor. Immunity 2007; 27(1)145–159
   PubMedGoogle Scholar
• Deaglio S, Dwyer KM, Gao W, et al. Adenosine generation catalyzed by CD39 and CD73 expressed on regulatory T cells mediates immune suppression. J Exp Med 2007; 204(6)1257–1265
   PubMed Web of Science ®Google Scholar
• McHugh RS, Shevach EM. The role of suppressor T cells in regulation of immune responses. J Allergy Clin Immunol 2002; 110(5)693–702
   PubMedGoogle Scholar
• Randolph DA, Fathman CG. Cd4+Cd25+ regulatory T cells and their therapeutic potential. Annu Rev Med 2006; 57: 381–402
   PubMed Web of Science ®Google Scholar
• Kyewski B, Derbinski J. Self-representation in the thymus: An extended view. Nat Rev Immunol 2004; 4(9)688–698
   PubMed Web of Science ®Google Scholar
• Kyewski B, Klein L. A central role for central tolerance. Annu Rev Immunol 2006; 24: 571–606
   PubMed Web of Science ®Google Scholar
• Cheng MH, Shum AK, Anderson MS. What's new in the Aire?. Trends Immunol 2007; 28(7)321–327
   PubMedGoogle Scholar
• Mathis D, Benoist C. A decade of AIRE. Nat Rev Immunol 2007; 7(8)645–650
   PubMed Web of Science ®Google Scholar
• Bjorses P, Aaltonen J, Horelli-Kuitunen N, et al. Gene defect behind APECED: A new clue to autoimmunity. Hum Mol Genet 1998; 7(10)1547–1553
   PubMedGoogle Scholar
• Peterson P, Nagamine K, Scott H, et al. APECED: A monogenic autoimmune disease providing new clues to self-tolerance. Immunol Today 1998; 19(9)384–386
   PubMedGoogle Scholar
• Anderson MS, Venanzi ES, Klein L, et al. Projection of an immunological self shadow within the thymus by the Aire protein. Science 2002; 298(5597)1395–1401
   PubMed Web of Science ®Google Scholar
• Klamp T, Sahin U, Kyewski B, et al. Expression profiling of autoimmune regulator AIRE mRNA in a comprehensive set of human normal and neoplastic tissues. Immunol Lett 2006; 106(2)172–179
   PubMedGoogle Scholar
• Kogawa K, Nagafuchi S, Katsuta H, et al. Expression of AIRE gene in peripheral monocyte/dendritic cell lineage. Immunol Lett 2002; 80(3)195–198
   PubMedGoogle Scholar
• Lee JW, Epardaud M, Sun J, et al. Peripheral antigen display by lymph node stroma promotes T cell tolerance to intestinal self. Nat Immunol 2007; 8(2)181–190
   PubMed Web of Science ®Google Scholar
• Liston A, Lesage S, Wilson J, et al. Aire regulates negative selection of organ-specific T cells. Nat Immunol 2003; 4(4)350–354
   PubMed Web of Science ®Google Scholar
• Liston A, Gray DH, Lesage S, et al. Gene dosage—limiting role of Aire in thymic expression, clonal deletion, and organ-specific autoimmunity. J Exp Med 2004; 200(8)1015–1026
   PubMedGoogle Scholar
• Kont V, Laan M, Kisand K, et al. Modulation of Aire regulates the expression of tissue-restricted antigens. Mol Immunol 2008; 45(1)25–33
   PubMedGoogle Scholar
• Taubert R, Schwendemann J, Kyewski B. Highly variable expression of tissue-restricted self-antigens in human thymus: Implications for self-tolerance and autoimmunity. Eur J Immunol 2007; 37(3)838–848
   PubMedGoogle Scholar
• Alderuccio F, Toh BH, Tan SS, et al. An autoimmune disease with multiple molecular targets abrogated by the transgenic expression of a single autoantigen in the thymus. J Exp Med 1993; 178(2)419–426
   PubMed Web of Science ®Google Scholar
• French MB, Allison J, Cram DS, et al. Transgenic expression of mouse proinsulin II prevents diabetes in nonobese diabetic mice. Diabetes 1997; 46(1)34–39
   PubMedGoogle Scholar
• Jaeckel E, Lipes MA, von Boehmer H. Recessive tolerance to preproinsulin 2 reduces but does not abolish type 1 diabetes. Nat Immunol 2004; 5(10)1028–1035
   PubMedGoogle Scholar
• Xu H, Wawrousek EF, Redmond TM, et al. Transgenic expression of an immunologically privileged retinal antigen extraocularly enhances self tolerance and abrogates susceptibility to autoimmune uveitis. Eur J Immunol 2000; 30(1)272–278
   PubMedGoogle Scholar
• Murphy K, Biondo M, Toh BH, Alderuccio F. Tolerance established in autoimmune disease by mating or bone marrow transplantation that target autoantigen to thymus. Int Immunol 2003; 15(2)269–277
   PubMedGoogle Scholar
• Steptoe RJ, Ritchie JM, Harrison LC. Transfer of hematopoietic stem cells encoding autoantigen prevents autoimmune diabetes. J Clin Invest 2003; 111(9)1357–1363
   PubMed Web of Science ®Google Scholar
• Billingham RE, Brent L, Medawar PB. Actively acquired tolerance of foreign cells. Nature 1953; 172(4379)603–606
   PubMed Web of Science ®Google Scholar
• Brocker T, Riedinger M, Karjalainen K. Targeted expression of major histocompatibility complex (MHC) class II molecules demonstrates that dendritic cells can induce negative but not positive selection of thymocytes in vivo. J Exp Med 1997; 185(3)541–550
   PubMed Web of Science ®Google Scholar
• Gallegos AM, Bevan MJ. Central tolerance: Good but imperfect. Immunol Rev 2006; 209: 290–296
   PubMed Web of Science ®Google Scholar
• Palmer E. Negative selection—clearing out the bad apples from the T-cell repertoire. Nat Rev Immunol 2003; 3(5)383–391
   PubMed Web of Science ®Google Scholar
• Yamazaki S, Inaba K, Tarbell KV, Steinman RM. Dendritic cells expand antigen-specific Foxp3+ CD25+ CD4+ regulatory T cells including suppressors of alloreactivity. Immunol Rev 2006; 212: 314–329
   PubMed Web of Science ®Google Scholar
• Yamazaki S, Patel M, Harper A, et al. Effective expansion of alloantigen-specific Foxp3+ CD25+ CD4+ regulatory T cells by dendritic cells during the mixed leukocyte reaction. Proc Natl Acad Sci USA 2006; 103(8)2758–2763
   PubMedGoogle Scholar
• van Laar JM, Tyndall A. Adult stem cells in the treatment of autoimmune diseases. Rheumatology (Oxford) 2006; 45(10)1187–1193
   PubMedGoogle Scholar
• Dazzi F, van Laar JM, Cope A, Tyndall A. Cell therapy for autoimmune diseases. Arthritis Res Ther 2007; 9(2)206
   PubMed Web of Science ®Google Scholar
• Sykes M, Nikolic B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature 2005; 435(7042)620–627
   PubMed Web of Science ®Google Scholar
• Nash RA, McSweeney PA, Crofford LJ, et al. High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: Long-term follow-up of the US multicenter pilot study. Blood 2007; 110(4)1388–1396
   PubMed Web of Science ®Google Scholar
• Alderuccio F, Murphy K, Toh BH. Stem cells engineered to express self-antigen to treat autoimmunity. Trends Immunol 2003; 24(4)176–180
   PubMed Web of Science ®Google Scholar
• Siatskas C, Chan J, Field J, et al. Gene therapy strategies towards immune tolerance to treat the autoimmune diseases. Curr Gene Ther 2006; 6(1)45–58
   PubMed Web of Science ®Google Scholar
• Ally BA, Hawley TS, McKall-Faienza KJ, et al. Prevention of autoimmune disease by retroviral-mediated gene therapy. J Immunol 1995; 155(11)5404–5408
   PubMed Web of Science ®Google Scholar
• Bagley J, Tian C, Sachs DH, Iacomini J. Induction of T-cell tolerance to an MHC class I alloantigen by gene therapy. Blood 2002; 99(12)4394–4399
   PubMedGoogle Scholar
• Tian C, Bagley J, Cretin N, et al. Prevention of type 1 diabetes by gene therapy. J Clin Invest 2004; 114(7)969–978
   PubMed Web of Science ®Google Scholar
• Tian C, Bagley J, Iacomini J. Persistence of antigen is required to maintain transplantation tolerance induced by genetic modification of bone marrow stem cells. Am J Transplant 2006; 6(9)2202–2207
   PubMedGoogle Scholar
• Bonilla WV, Geuking MB, Aichele P, et al. Microchimerism maintains deletion of the donor cell-specific CD8+T cell repertoire. J Clin Invest 2006; 116(1)156–162
   PubMedGoogle Scholar
• Chan J, Clements W, Field J, et al. Transplantation of bone marrow genetically engineered to express proinsulin II protects against autoimmune insulitis in NOD mice. J Gene Med 2006; 8(11)1281–1290
   PubMedGoogle Scholar
• Xu B, Haviernik P, Wolfraim LA, et al. Bone marrow transplantation combined with gene therapy to induce antigen-specific tolerance and ameliorate EAE. Mol Ther 2006; 13(1)42–48
   PubMedGoogle Scholar
• Kang ES, Iacomini J. Induction of central deletional T cell tolerance by gene therapy. J Immunol 2002; 169(4)1930–1935
   PubMedGoogle Scholar
• Forman D, Kang ES, Tian C, et al. Induction of alloreactive CD4 T cell tolerance in molecular chimeras: A possible role for regulatory T cells. J Immunol 2006; 176(6)3410–3416
   PubMedGoogle Scholar
• Tian C, Bagley J, Iacomini J. Homeostatic expansion permits T cells to re-enter the thymus and deliver antigen in a tolerogenic fashion. Am J Transplant 2007; 7(8)1934–1941
   PubMedGoogle Scholar
• Berns A. Good news for gene therapy. N Engl J Med 2004; 350(16)1679–1680
   PubMed Web of Science ®Google Scholar
• Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003; 348(3)255–256
   PubMed Web of Science ®Google Scholar