Gene therapy and bone marrow stem-cell transfer to treat autoimmune disease

Trends in Molecular Medicine

Volumn 15, Issue 8, 2009, Pages 344-351

  Alderuccio, Frank ^a   Chan, James ^b   Scott, David W ^c   Toh, Ban Hock ^b  

^a MONASH UNIVERSITY   (Australia)

^b MONASH MEDICAL CENTRE   (Australia)

^c UNIVERSITY OF MARYLAND SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALLERGIC ENCEPHALOMYELITIS; ARTICLE; AUTOIMMUNE DISEASE; AUTOIMMUNE UVEITIS; CELL TRANSFER; GENE THERAPY; GENETIC MANIPULATION; HEMATOPOIETIC STEM CELL; HUMAN; IMMUNOLOGICAL TOLERANCE; INSULIN DEPENDENT DIABETES MELLITUS; NONHUMAN; UVEITIS;

ANIMALS; AUTOIMMUNE DISEASES; BONE MARROW TRANSPLANTATION; GENE THERAPY; HUMANS; STEM CELL TRANSPLANTATION;

EID: 68949125744     PISSN: 14714914     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.molmed.2009.06.002     Document Type: Article

References (85)

1. Rose N.R., and Mackay I.R. The Autoimmune Diseases (2006), Elsevier Academic Press
2. Owen R.D. Immunogenetic consequences of vascular anastomoses between bovine twins. Science 102 (1945) 400-401
3. Billingham R.E., et al. Activity acquired tolerance of foreign cells. Nature 172 (1953) 603-606
4. Chernajovsky Y., et al. Gene therapy for autoimmune diseases: quo vadis?. Nat. Rev. Immunol. 4 (2004) 800-811
5. Furlan R., et al. Gene therapy for autoimmune diseases. Curr. Opin. Mol. Ther. 6 (2004) 525-536
6. Siatskas C., et al. Gene therapy strategies towards immune tolerance to treat the autoimmune diseases. Curr. Gene Ther. 6 (2006) 45-58
7. Deane S., et al. On reversing the persistence of memory: hematopoietic stem cell transplant for autoimmune disease in the first ten years. J. Autoimmun. 30 (2008) 180-196
8. Marmont A.M. Will heamatopoietic stem cell transplantation cure human autoimmune diseases?. J. Autoimmun. 30 (2008) 145-150
9. Sykes M., and Nikolic B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature 435 (2005) 620-627
10. Saccardi R., and Gualandi F. Hematopoietic stem cell transplantation procedures. Autoimmunity 41 (2008) 570-576
11. van Laar J.M., et al. Stem cell transplantation: a treatment option for severe systemic sclerosis?. Ann. Rheum. Dis. 67 Suppl 3 (2008) iii35-iii38
12. Burt R.K., et al. Autologous non-myeloablative haemopoietic stem cell transplantation in relapsing-remitting multiple sclerosis: a phase I/II study. Lancet Neurol. 8 (2009) 244-253
13. Alaez C., et al. Hematopoietic stem cell transplantation (HSCT): an approach to autoimmunity. Autoimmun. Rev. 5 (2006) 167-179
14. Tyndall A., and Le Blanc K. Stem cell transplantation for autoimmune disease. Cellular Transplantation: From Laboratory to Clinic (2007), Elsevier pp. 241-257
15. Fassas A., and Mancardi G.L. Autologous hemopoietic stem cell transplantation for multiple sclerosis: is it worthwhile?. Autoimmunity 41 (2008) 601-610
16. Burt R.K., et al. Hematopoietic stem cell transplantation for autoimmune diseases: What have we learned?. J. Autoimmun. 30 (2008) 116-120
17. Marmont A.M., and Saccardi R. Concluding remarks. Autoimmunity 41 (2008) 686-690
18. de Kleer I., et al. Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood 107 (2006) 1696-1702
19. Alexander T., et al. Depletion of autoreactive immunologic memory followed by autologous hematopoietic stem cell transplantation in patients with refractory SLE induces long-term remission through de novo generation of a juvenile and tolerant immune system. Blood 113 (2009) 214-223
20. Douek D.C., and Koup R.A. Evidence for thymic function in the elderly. Vaccine 18 (2000) 1638-1641
21. Muraro P.A., et al. Thymic output generates a new and diverse TCR repertoire after autologous stem cell transplantation in multiple sclerosis patients. J. Exp. Med. 201 (2005) 805-816
22. Hershfield M.S. Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy. Semin. Hematol. 35 (1998) 291-298
23. Leonard W. Type 1 cytokines and interferons and their receptors. In: Paul W.E. (Ed). Fundamental Immunology. 5th edn (2003), Lippincott Williams and Wilkins 701-747
24. Aiuti A., et al. Gene therapy for immunodeficiency due to adenosinen deaminase deficiency. N. Engl. J. Med. 360 (2009) 447-458
25. Hacein-Bey-Abina S., et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N. Engl. J. Med. 346 (2002) 1185-1193
26. Gaspar H.B., et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped γretroviral vector. Lancet 364 (2004) 2181-2187
27. Hacein-Bey-Abina S., et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348 (2003) 255-256
28. Hacein-Bey-Abina S., et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (2003) 415-419
29. Berns A. Good news for gene therapy. N. Engl. J. Med. 350 (2004) 1679-1680
30. Kohn D.B., and Condotti F. Gene therapy fulfilling its promise. N. Engl. J. Med. 360 (2009) 518-521
31. Yi Y., et al. Retroviral gene therapy: safety issues and possible solutions. Curr. Gene Ther. 5 (2005) 25-35
32. Wenner M. Regaining lost luster. Sci. Am. (January) (2007) 18-19
33. Wagstaff K.M., et al. Efficient gene delivery using reconstituted chromatin enhanced for nuclear targeting. FASEB J. 22 (2008) 2232-2242
34. Glover D.J., et al. Towards safe, non-viral therapeutic gene expression in humans. Nat. Rev. Genet. 6 (2005) 299-310
35. Melo M.E., et al. Gene transfer of Ig-fusion proteins into B cells prevents and treats autoimmune diseases. J. Immunol. 168 (2002) 4788-4795
36. De Groot A.S., et al. Activation of natural regulatory T cells by IgG Fc-derived peptide "Tregitopes". Blood 112 (2008) 3303-3311
37. Su Y., et al. B cells induce tolerance by presenting endogenous peptide-IgG on MHC class II molecules via an IFN-gamma-inducible lysosomal thiol reductase-dependent pathway. J. Immunol. 181 (2008) 1153-1160
38. Alderuccio F., et al. An autoimmune disease with multiple molecular targets abrogated by the transgenic expression of a single autoantigen in the thymus. J. Exp. Med. 178 (1993) 419-426
39. French M.B., et al. Transgenic expression of mouse proinsulin II prevents diabetes in nonobese diabetic mice. Diabetes 46 (1997) 34-39
40. Xu H., et al. Transgenic expression of an immunologically privileged retinal antigen extraocularly enhances self tolerance and abrogates susceptibility to autoimmune uveitis. Eur. J. Immunol. 30 (2000) 272-278
41. Murphy K., et al. Tolerance established in autoimmune disease by mating or bone marrow transplantation that target autoantigen to thymus. Int. Immunol. 15 (2003) 269-277
42. Steptoe R.J., et al. Transfer of hematopoietic stem cells encoding autoantigen prevents autoimmune diabetes. J. Clin. Invest. 111 (2003) 1357-1363
43. Ally B.A., et al. Prevention of autoimmune disease by retroviral-mediated gene therapy. J. Immunol. 155 (1995) 5404-5408
44. Tian C., et al. Prevention of type 1 diabetes by gene therapy. J. Clin. Invest. 114 (2004) 969-978
45. Xu B., et al. Bone marrow transplantation combined with gene therapy to induce antigen-specific tolerance and ameliorate EAE. Mol. Ther. 13 (2006) 42-48
46. Chan J., et al. Transplantation of bone marrow transduced to express self-antigen establishes deletional tolerance and permanently remits autoimmune disease. J. Immunol. 181 (2008) 7571-7580
47. Chan J., et al. Transplantation of bone marrow genetically engineered to express proinsulin II protects against autoimmune insulitis in NOD mice. J. Gene Med. 8 (2006) 1281-1290
48. Baranyi U., et al. Tolerization of a type 1 allergic response through transplantation of genetically modified hematopoietic stem cells. J. Immunol. 180 (2008) 8168-8175
49. Dresch C., et al. Lentiviral-mediated transcriptional targeting of dendritic cells for induction of T cell tolerance in vivo. J. Immunol. 181 (2008) 4495-4506
50. Biondo M., et al. Prednisolone promotes remission and gastric mucosal regeneration in experimental autoimmune gastritis. J. Pathol. 209 (2006) 384-391
51. Chan J., et al. Methylprednisolone induces reversible clinical and pathological remission and loss of lymphocyte reactivity to myelin oligodendrocyte glycoprotein in experimental autoimmune encephalomyelitis. Autoimmunity 41 (2008) 405-413
52. Anderson M.S., et al. Projection of an immunological self shadow within the thymus by the aire protein. Science 298 (2002) 1395-1401
53. Liston A., et al. Aire regulates negative selection of organ-specific T cells. Nat. Immunol. 4 (2003) 350-354
54. Kang E.S., and Iacomini J. Induction of central deletional T cell tolerance by gene therapy. J. Immunol. 169 (2002) 1930-1935
55. Bettelli E., et al. Myelin oligodendrocyte glycoprotein-specific T cell receptor transgenic mice develop spontaneous autoimmune optic neuritis. J. Exp. Med. 197 (2003) 1073-1081
56. Tian C., et al. Persistence of antigen is required to maintain transplantation tolerance induced by genetic modification of bone marrow stem cells. Am. J. Transplant. 6 (2006) 2202-2207
57. Wekerle T., and Sykes M. Mixed chimerism and transplantation tolerance. Annu. Rev. Med. 52 (2001) 353-370
58. Hayden P.J., et al. Use of DLI to achieve complete donor chimerism in a patient receiving systemic immunosuppression for refractory AIHA post-NST. Bone Marrow Transplant. 36 (2005) 735-736
59. Musso M., et al. Donor lymphocyte infusions for refractory pure red cell aplasia relapsing after both autologous and nonmyeloablative allogeneic peripheral stem cell transplantation. Bone Marrow Transplant. 33 (2004) 769-771
60. Xu B., and Scott D.W. A novel retroviral gene therapy approach to inhibit specific antibody production and suppress experimental autoimmune encephalomyelitis induced by MOG and MBP. Clin. Immunol. 111 (2004) 47-52
61. + regulatory T cells and TGF-β?. Gene Ther. 11 (2004) 1487-1496
62. + regulatory T cells are responsible for GAD-IgG gene-transferred tolerance induction in NOD mice. Immunology 126 (2009) 123-131
63. Hirata S., et al. Prevention of experimental autoimmune encephalomyelitis by transfer of embryonic stem cell-derived dendritic cells expressing myelin oligodendrocyte glycoprotein peptide along with TRAIL or programmed death-1 ligand. J. Immunol. 174 (2005) 1888-1897
64. Agarwal R.K., et al. Retroviral gene therapy with an immunoglobulin-antigen fusion construct protects from experimental autoimmune uveitis. J. Clin. Invest. 106 (2000) 245-252
65. Mathisen P.M., et al. Treatment of experimental autoimmune encephalomyelitis with genetically modified memory T cells. J. Exp. Med. 186 (1997) 159-164
66. Tarner I.H., and Fathman C.G. The potential for gene therapy in the treatment of autoimmune disease. Clin. Immunol. 104 (2002) 204-216
67. Yamamoto A.M., et al. The activity of immunoregulatory T cells mediating active tolerance is potentiated in nonobese diabetic mice by an IL-4-based retroviral gene therapy. J. Immunol. 166 (2001) 4973-4980
68. Shaw M.K., et al. Local delivery of interleukin 4 by retrovirus-transduced T lymphocytes ameliorates experimental autoimmune encephalomyelitis. J. Exp. Med. 185 (1997) 1711-1714
69. + hybridoma cells in experimental autoimmune encephalomyelitis. Eur. Cytokine Netw. 9 3 Suppl (1998) 83-91
70. Morita Y., et al. Dendritic cells genetically engineered to express IL-4 inhibit murine collagen-induced arthritis. J. Clin. Invest. 107 (2001) 1275-1284
71. Chen L.Z., et al. Gene therapy in allergic encephalomyelitis using myelin basic protein-specific T cells engineered to express latent transforming growth factor-beta1. Proc. Natl. Acad. Sci. U. S. A. 95 (1998) 12516-12521
72. Zargarova T., et al. Prevention of experimental autoimmune encephalomyelitis in DA rats by grafting primary skin fibroblasts engineered to express transforming growth factor-β1. Clin. Exp. Immunol. 137 (2004) 313-319
73. Jaeckel E., et al. Antigen-specific FoxP3-transduced T-cells can control established type 1 diabetes. Diabetes 54 (2005) 306-310
74. Peng J., et al. Converting antigen-specific diabetogenic CD4 and CD8 T cells to TGF-β producing non-pathogenic regulatory cells following FoxP3 transduction. J. Autoimmun. 28 (2007) 188-200
75. - T cells by in vitro Foxp3 gene transfer, can induce transplantation tolerance. Transplantation 79 (2005) 1310-1316
76. + T cells under the control of IFN-g promoter prevent diabetes in NOD mice. Mol. Ther. 15 (2007) 1551-1557
77. Gallegos A.M., and Bevan M.J. Central tolerance to tissue-specific antigens mediated by direct and indirect antigen presentation. J. Exp. Med. 200 (2004) 1039-1049
78. Kyewski B., and Klein L. A central role for central tolerance. Annu. Rev. Immunol. 24 (2006) 571-606
79. Bonasio R., et al. Clonal deletion of thymocytes by circulating dendritic cells homing to the thymus. Nat. Immunol. 7 (2006) 1092-1100
80. Proietto A.I., et al. Dendritic cells in the thymus contribute to T-regulatory cell induction. Proc. Natl. Acad. Sci. U. S. A. 105 (2008) 19869-19874
81. Ohnmacht C., et al. Constitutive ablation of dendritic cells breaks self-tolerance of CD4 T cells and results in spontaneous fatal autoimmunity. J. Exp. Med. 206 (2009) 549-559
82. Kyewski B., and Derbinski J. Self-representation in the thymus: an extended view. Nat. Rev. Immunol. 4 (2004) 688-698
83. Gray D., et al. Proliferative arrest and rapid turnover of thymic epithelial cells expressing Aire. J. Exp. Med. 204 (2007) 2521-2528
84. Peterson P., et al. APECED: a monogenic autoimmune disease providing new clues to self-tolerance. Immunol. Today 19 (1998) 384-386
85. Liston A., et al. Gene dosage-limiting role of Aire in thymic expression, clonal deletion, and organ-specific autoimmunity. J. Exp. Med. 200 (2004) 1015-1026