Gene therapy of autoimmune diseases with vectors encoding regulatory cytokines or inflammatory cytokine inhibitors

Journal of Gene Medicine

Volumn 2, Issue 4, 2000, Pages 222-232

  Prud'homme, Gérald J ^a  

^a MCGILL UNIVERSITY   (Canada)

Author keywords

Autoimmunity; Cytokine; Gene therapy; Interferon; Interleukin 1 receptor antagonist; Naked DNA; T lymphocytes; Transforming growth factor beta; Tumor necrosis factor

Indexed keywords

ANIMALIA;

CYTOKINE;

ANIMAL; AUTOIMMUNE DISEASE; DENDRITIC CELL; DRUG ANTAGONISM; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; IMMUNOLOGY; IMMUNOTHERAPY; INSULIN DEPENDENT DIABETES MELLITUS; REVIEW; RHEUMATOID ARTHRITIS;

ANIMALS; ARTHRITIS, RHEUMATOID; AUTOIMMUNE DISEASES; CYTOKINES; DENDRITIC CELLS; DIABETES MELLITUS, TYPE 1; GENE THERAPY; GENETIC VECTORS; HUMANS; IMMUNOTHERAPY;

EID: 0034220667     PISSN: 1099498X     EISSN: None     Source Type: Journal    
DOI: 10.1002/1521-2254(200007/08)2:4<222::aid-jgm117>3.0.co;2-p     Document Type: Review

References (98)

1. Trembleau S, Germann T, Gately MK, Adorini L. The role of IL-12 in the induction of organ-specific autoimmune diseases. Immunol Today 1995; 16: 383-386.
2. Liblau RS, Singer SM, McDevitt HO. Th1 and Th2 CD4+ T cells in the pathogenesis of organ-specific autoimmune diseases. Immunol Today 1995; 16: 34-38.
3. Kalden JR, Breedveld FC, Burkhardt H, Burmester GR. Immunological treatment of autoimmune diseases. Adv Immunol 1998; 68: 333-418.
4. Rabinovitch A. An update on cytokines in the pathogenesis of Type 1 diabetes mellitus. Diabetes Metab Rev 1998; 14: 129-151.
5. Delovitch TL, Singh B. The nonobese diabetic mouse as a model of autoimmune diabetes: immune dysregulation gets the NOD. Immunity 1997; 7: 728-738.
6. Katz JD, Benoist C, Mathis D. T helper subsets in Type 1 diabetes. Science 1995; 268: 1185-1188.
7. Prud'homme GJ, Chang Y. Prevention of autoimmune diabetes by intramuscular gene therapy with a nonviral vector encoding an interferon-gamma receptor/IgG1 fusion protein. Gene Ther 1999; 6: 771-777.
8. Wang D, Andre I, Gonzalez A, Katz JD, Aguet M, Benoist C, Mathis D. Interferon-gamma impacts at multiple points during the progression of autoimmune diabetes. Proc Natl Acad Sci U S A 1997; 94: 13844-13849.
9. Trembleau S, Penna G, Gregori S, Gately MK, Adorini L. Deviation of pancreas infiltrating cells to Th2 by interleukin 12 antagonists administration inhibits autoimmune diabetes. Eur J Immunol 1997; 27: 2330-2339.
10. Grewall IS, Flavell RA. New insights into Type 1 diabetes mellitus from studies with transgenic mouse models. Lab Invest 1997; 76: 3-10.
11. Chang Y, Prud'homme GJ. Intramuscular administration of expression plasmids encoding interferon-gamma-receptor/IgG1 or IL-4/IgG1 chimeric proteins protects from autoimmunity. J Gene Med 1999; 1: 415-423.
12. Romagnani S. Th1/Th2 cells. Inflammatory Bowel Dis 1999; 5: 285-294.
13. Weinberg AD, Whitman R, Swain SL, Morrison WJ, Wyrick G, Hoy C, Vandenbark AA, Offner H. Target organ-specific up-regulation of the MRC OX-40 marker and selective production of Th1 lymphokine mRNA by encephalitogenic T helper cells isolated from the spinal cord of rats with EAE. J Immunol 1992; 148: 2109-2117.
14. Olsson T. Critical influences of the cytokine orchestration on the outcome of myelin antigen-specific T cell autoimmunity in experimental allergic encephalomyelitis and multiple sclerosis. Immunol Rev 1995; 144: 245-268.
15. Swanborg RH. Experimental autoimmune encephalomyelitis in rodents as a model for human demyelinating disease. Clin Immunol Immunopathol 1995; 77: 4-13.
16. Boyle DL, Nguyen KHY, Zhuang S, Shi Y, McCormack JE, Firestein GS. Intra-articular IL-4 gene therapy in arthritis: anti-inflammatory effect and enhanced Th2 activity. Gene Ther 1999; 6: 1911-1918.
17. Bigazzi PE. Animal models of autoimmunity: spontaneous and induced. In The Autoimmune Diseases (3rd edn), Rose NR, Mackey IR (eds). Academic Press: San Diego, USA, 1998; 211-244.
18. Prud'homme GJ, Piccirillo CA. The inhibitory effects of transforming growth factor beta-1 (TGF-β1) in autoimmune diseases. J Autoimmun 2000; 14: 23-42.
19. Derynck R, Choy L. Transforming growth factor-β and its receptors. In The Cytokine Handbook (3rd edn), Thompson A (ed.). Academic Press: New York, 1998; 593-638.
20. Ruscetti FW, Birchenall-Roberts MC, McPherson JM, Wiltrout RH. Transforming growth factor β1. In Cytokines, Mire-Luis A, Thorpe R (eds). Academic Press: San Diego, USA, 1998; 415-432.
21. Letterio JJ, Roberts AB. Regulation of immune responses by TGF-β. Annu Rev Immunol 1998; 6: 137-161.
22. McCartney-Francis NL, Frazier-Jessen M, Wahl SM. TGF-beta: a balancing act. Int Rev Immunol 1998; 16: 553-580.
23. Engel ME, Datta PK, Moses HL. Signal transduction by transforming growth factor-beta: a cooperative paradigm with extensive negative regulation. J Cell Biochem Suppl 1998; 31: 111-122.
24. Faria AM, Weiner HL. Oral tolerance: mechanisms and therapeutic applications. Adv Immunol 1999; 73: 153-264.
25. Kallapur S, Shull M, Doetschman T. Phenotypes of TGFβ knockout mice. In Cytokine Knockouts, Durum SK, Mueege K (eds). Humana Press: Totowa, NJ, 1998; 315-334.
26. Kulkarni AB, Letterio JJ. The transforming growth factor-β1 knockout mouse: The phenotype and its implications for TGFβ1 function. In Cytokine Knockouts, Durum SK, Mueege K (eds). Humana Press: Totowa, NJ, 1998; 369-400.
27. Bessis N, Boissier MC, Ferrara P, Blankenstein T, Fradelizi D, Fournier C. Attenuation of collagen-induced arthritis in mice by treatment with vector cells engineered to secrete interleukin-13. Eur J Immunol 1996; 26: 2399-2405.
28. Bessis N, Honiger J, Damotte D, et al. Encapsulation in hollow fibres of xenogeneic cells engineered to secrete IL-4 or IL-13 ameliorates murine collagen-induced arthritis. Clin Exp Immunol 1999; 117: 376-382.
29. Prud'homme GJ, Kono DH. Theofilopoulos AN. Quantitative polymerase chain reaction analysis reveals marked overexpression of interleukin-1β, interleukin-10, and interferon-γ mRNA in the lymph nodes of lupus-prone mice. Mol Immunol 1995; 32: 495-503.
30. Wallick SC, Figari IS , Morris RE, Levison AD, Palladino MA. Immunoregulatory role of transforming growth factor beta (TGF-beta) in development of killer cells: Comparison of active and latent TGF-beta1. J Exp Med 1990; 172: 1777-1784.
31. Kuruvilla AP, Shah R, Hochwald GM, Liggitt HD, Palladino MA, Thorbecke GJ. Protective effect of transforming growth factor β1 on experimental autoimmune disease in mice. Proc Natl Acad Sci U S A 1991; 88: 2918-2921.
32. Lennart DJ, Flanders KC, Rangers GE, Sriram S. Successful treatment of experimental allergic encephalomyelitis with transforming growth factor-β1. J Immunol 1991; 147: 1792-1796.
33. 1. J Immunol 1991; 146: 3012-3017.
34. Piccirillo C, Chang Y, Prud'homme GJ. Transforming growth factor beta-1 (TGF-β1) somatic gene therapy prevents autoimmune disease in NOD mice. J Immunol 1998; 161: 3950-3956.
35. Raz E, Watanabe A, Baird SM, et al. Systemic immunological effects of cytokine genes injected into skeletal muscle. Proc Natl Acad Sci U S A 1993; 90: 4523-4527.
36. Cameron MJ, Arreaza GA, Zucker P, et al. IL-4 prevents insulitis and insulin-dependent diabetes mellitus in nonobese diabetic mice by potentiation of regulatory T-helper-2 cell function. J Immunol 1997; 159: 4686-4692.
37. Mueller R, Krahl T, Sarvetnick N. Pancreatic expression of interleukin-4 abrogates insulitis and autoimmune diabetes in nonobese diabetic (NOD) mice. J Exp Med 1996; 184: 1093-1099.
38. Mueller R, Bradley LM, Krahl T, Sarvetnick N. Mechanism underlying counterregulation of autoimmune diabetes by IL-4. Immunity 1997; 7: 411-418.
39. Hartikka J, Sawdey M, Cornefert-Jensen F, et al. An improved plasmid DNA expression vector for direct injection into skeletal muscle. Hum Gene Ther 1996; 7: 1205-1217.
40. Ishii KJ, Weiss WR, Ichino M, Verthelyi D, Klinman DM. Activity and safety of DNA plasmids encoding IL-4 and IFN gamma. Gene Ther 1999; 6: 237-244.
41. Piccirillo C, Prud'homme GJ. Prevention of experimental allergic encephalomyelitis by intramuscular gene therapy with cytokine-encoding plasmid vectors. Hum Gene Ther 1999; 10: 1915-1922.
42. Croxford JL, Triantaphyllapoulos K, Podhajcer LL, Feldmann M, Baker D, Chernajovsky Y. Cytokine gene therapy in experimental allergic encephalomyelitis by injection of plasmid DNA-cationic liposome complex into the central nervous system. J Immunol 1998; 160: 5181-5187.
43. Nitta Y, Tashiro F, Tokui M, et al. Systemic delivery of interleukin 10 by intramuscular injection of expression plasmid DNA prevents autoimmune diabetes in nonobese diabetic mice. Hum Gene Ther 1998; 9: 1701-1707.
44. Raz E, Duddler J, Lotz M, Bairs SM, Berry CC, Eisenberg RA, Carson D. Modulation of disease activity in murine systemic lupus erythematosus by cytokine gene delivery. Lupus 1995; 4: 286-292.
45. Giladi E, Raz E, Karmeli F, Okon E, Rachmilewitz D. Transforming growth factor-beta gene therapy ameliorates experimental colitis in rats. Eur J Gastroenterol Hepatol 1995; 7: 341-347.
46. Song X-Y, Mili G, Jin WW, Klinman DM, Wahl SM. Plasmid DNA encoding transforming growth factor-β1 suppresses chronic disease in a streptococcal cell wall-induced arthritis model. J Clin Invest 1998; 101: 2615-2621.
47. Chen LZ, Hochwald GM, Huang C, et al. Gene therapy in allergic encephalomyelitis using myelin basic protein-specific T cells engineered to express latent transforming growth factor-beta 1. Proc Natl Acad Sci U S A 1998; 95: 12516-12521.
48. Dal Canto RA, Costa G, Shaw MD, Seroogy C, Nolan GP, Fathman CG. Local delivery of cytokines by retrovirally transduced antigen-specific TCR+ hybridoma cells in experimental autoimmune encephalomyelitis. Eur Cytokine Netw 1998; 9: 83-91.
49. Shaw MK, Lorens JB, Dhawan A, et al. Local delivery of interleukin 4 by retrovirus-transduced T lymphocytes ameliorates experimental autoimmune encephalomyelitis. J Exp Med 1997; 185: 1711-1714.
50. Moritani M, Yoshimoto K, Li S, et al. Prevention of adoptively. transferred diabetes in nonobese diabetic mice with IL-10-transduced islet-specific Th1 lymphocytes. A gene therapy model for autoimmune diabetes. J Clin Invest 1996; 98: 1851-1859.
51. Batteux F, Trebeden H, Charreire J, Chiocchia G. Curative treatment of experimental autoimmune thyroiditis by in vivo administration of plasmid DNA coding for interleukin-10. Eur J Immunol 1999; 29: 958-963.
52. Whalen JD, Lechman EL, Carlos CA, et al. Adenoviral transfer of the viral IL-10 gene periarticularly to mouse paws suppresses development of collagen-induced arthritis in both injected and uninjected paws. J Immunol 1999; 162: 3625-3632.
53. Yasuda H, Nagata M, Arisawa K, et al. Local expression of immunoregulatory IL-12p40 gene prolonged syngeneic islet graft survival in diabetic NOD mice. J Clin Invest 1998; 102: 1807-1814.
54. Chun S, Daheshia M, Chun S, Daheshia M, Lee S, Rouse BT. Immune modulation by IL-10 gene transfer via viral vector and plasmid DNA: Implication for gene therapy. Cell Immunol 1999; 194: 194-204.
55. Krieg AM. Direct immunologic activities of CpG DNA and implications for gene therapy. J Gene Med 1999; 1: 56-63.
56. Mir LM, Bureau MF, Gehl J, et al. High-efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc Natl Acad Sci U S A 1999; 96: 4262-4267.
57. Mathiesen I. Electropermeabilization of skeletal muscle enhances gene transfer in vivo. Gene Ther 1999; 6: 508-514.
58. Kreiss P, Bettan M, Crouzet J, Scherman D. Erythropoietin secretion and physiological effect in mouse after intramuscular plasmid DNA electrotransfer. J Gene Med 1999; 1: 245-250.
59. Mir LM, Bureau MF, Rangara R, Schwartz B, Scherman D. Long-term, high level in vivo gene expression after electric pulse-mediated gene transfer into skeletal muscle. C R Acad Sci III 1998; 321: 893-899.
60. Harrison RL, Byrne BJ, Tung L. Electroporation-mediated gene transfer in cardiac tissue. FEBS Lett 1998; 435: 1-5.
61. Suzuki T, Shin BC, Fujikura K, Matsuzaki T, Takata K. Direct gene transfer into rat liver cells by in vivo electroporation. FEBS Lett 1998; 425: 436-440.
62. Nishi T, Yoshizato K, Yamashiro S, et al. High-efficiency in vivo gene transfer using intraarterial plasmid DNA injection following in vivo electroporation. Cancer Res 1996; 56: 1050-1055.
63. Neumann E, Kakorin S, Toensing K. Fundamentals of electroporative delivery of drugs and genes. Bioelectrochem Bioenerg 1999; 48: 3-16.
64. Singh BN, Dwivedi C. Antitumor drug delivery by tissue electroporation. Anticancer Drugs 1999; 10: 139-146.
65. Mir LM, Touneki O, Orlowski S. Bleomycin: revival of an old drug. Gen Pharmacol 1996; 27: 745-748.
66. Nicoletti F, Zacone P, Di Marco R, et al. The effects of a nonimmunogenic form of murine soluble interferon-gamma receptor on the development of autoimmune diabetes in the NOD mouse. Endocrinology 1996; 137: 5567-5575.
67. Kürschner C, Ozmen L, Garotta G, Dembic Z. IFN-gamma receptor-Ig fusion proteins. Half-life, immunogenicity and in vivo activity. J Immunol 1992; 144: 4096-4100.
68. Campbell IL, Kay TW, Oxbrow L, Harrison LC. Essential role for interferon-gamma and interleukin-6 in autoimmune Type 1 diabetes in NOD/WEHI mice. J Clin Invest 1991; 87: 739-742.
69. Rothe H, Faust A, Schade U, et al. Cyclophosphamide treatment of female NOD mice causes enhanced expression of inducible nitric oxide synthase and interferon-gamma, but not IL-4. Diabetologia 1994; 37: 1154-1158.
70. Sarvetnick N, Shizuru J, Liggitt D, et al. Loss of pancreatic islet tolerance induced by beta-cell expression of interferon-gamma. Nature 1990; 346: 844-847.
71. Harms LS, Splitter GA. Interferon-gamma inhibits transgene expression driven by SV40 or CMV promoters but augments expression driven by the mammalian MHC 1 promoter. Hum Gene Ther 1995; 6: 1291-1297.
72. Hultgren B, Huang X, Dybdal N, Stewart TA. Genetic absence of gamma-interferon delays but does not prevent diabetes in NOD mice. Diabetes 1996; 45: 812-817.
73. Wang B, Andre I, Gonzalez A, et al. Interferon-gamma impacts at multiple points during the progression of autoimmune diabetes. Proc Natl Acad Sci U S A 1997; 94: 13844-13849.
74. Haak-Frendscho M, Marsters SA, Chamow SM, Peers DH, Simpson NJ, Ashkenazi A. Inhibition of interferon-gamma by an interferon-gamma receptor immunoadhesin. Immunology 1993; 79: 594-599.
75. Kürschner C, Garotta G, Dembic Z. Construction, Purification, and characterization of new interferon gamma inhibitor proteins. J Biol Chem 1992; 267: 9354-9360.
76. Makarov SS, Olsen JC, Johnston WN, et al. Suppression of experimental arthritis by gene transfer of interleukin 1 receptor antagonist cDNA. Proc Natl Acad Sci U S A 1996; 93: 402-406.
77. Bakker AC, Joosten LA, Arntz OJ, et al. Prevention of murine collagen-induced arthritis in the knee and ipsilateral paw by local expression of human interleukin-1 receptor antagonist (HuIL-1Ra) gene therapy in murine collagen-induced arthritis (CIA). Arthritis Rheum 1997; 40: 893-900.
78. Evans CH, Ghivizzani SC, Robbins PD. Blocking cytokines with genes. J Leukocyte Biol 1998; 64: 55-61.
79. Evans CH, Whalen JD, Ghivizzani SC, Robbins PD. Gene therapy in autoimmune diseases. Ann Rheum Dis 1998; 57: 125-135.
80. Muller-Ladner U, Roberts CR, Franklin BN, et al. Human IL-1Ra gene transfer into human synovial fibroblasts is chondroprotective. J Immunol 1997; 158: 3492-3498.
81. Robbins PD, Evans CH. Arthritis gene therapy. Drugs Today 1999; 35: 353-360.
82. Evans CH, Robbins PD. Gene therapy of arthritis. Int Med 1999; 38: 233-239.
83. Ghivizzani SC, Lechman ER, Kang R, et al. Direct adenovirus-mediated gene transfer of interleukin 1 and tumor necrosis factor alpha soluble receptors to rabbit knees with experimental arthritis has local and distal anti-arthritic effects. Proc Natl Acad Sci U S A 1998; 95: 4613-4618.
84. Pelletier JP, Caron JP, Evans C, et al. In vivo suppression of early experimental osteoarthritis by interleukin-1 receptor antagonist using gene therapy. Arthritis Rheum 1997; 40: 1012-1019.
85. Otani K, Nita I, Macaulay W, Georgescu HI, Robbins PD, Evans CH. Suppression of antigen-induced arthritis in rabbits by ex vivo gene therapy. J Immunol 1996; 156: 3558-3562.
86. Fernandes J, Tardif G, Martel-Pelletier J, et al. In vivo transfer of interleukin-1 receptor antagonist gene in osteoarthritic rabbit knee joints - Prevention of osteoarthritis progression. Am J Pathol 1999; 154: 1159-1169.
87. Quattrocchi E, Walmsley M, Browne K, et al. Paradoxical effects of adenovirus-mediated blockade of TNF activity in murine collagen-induced arthritis. J Immunol 1999; 163: 1000-1009.
88. Kim KN, Watanabe S, Ma Y, Thornton S, Giannini EH, Hirsch R. Viral IL-10 and soluble TNF receptor act synergistically to inhibit collagen-induced arthritis following adenovirus-mediated gene transfer. J Immunol 2000; 164: 1576-1581.
89. Day CS, Kasemkijwattana C, Menetrey J, et al. Myoblast-mediated gene transfer to the joint. J Orthop Res 1997; 15: 894-903.
90. Ludewig B, Odermatt B, Oschsenhein AF, Zinkernagel RM, Hengartner H. Role of dendritic cells in the induction and maintenance of autoimmune diseases. Immunol Rev 1999; 169: 45-54.
91. Bell B, Young JW, Banchereau J. Dendritic cells. Adv Immunol 1999; 72: 255-324.
92. Kalinki P, Hilkens CMU, Wierenga EA, Kapsenberg ML. T-cell priming by type-1 and type-2 polarized dendritic cells: the concept of a third signal. Immunol Today 1999; 20: 561-567.
93. Lu L, Lee W-C, Takayama T, et al. Genetic engineering of dendritic cells to express immunosuppressive molecules (viral IL-10, TGF-beta, and CTLA4Ig). J Leukoc Biol 1999; 66: 293-296.
94. Crystal RG. In vivo and ex vivo gene therapy strategies to treat tumors using adenovirus gene transfer vectors. Cancer Chemother Pharmacol 1999; 43 (Suppl.): S90-99.
95. Van Tendeloo VF, Snoeck HW, Lardon F, et al. Nonviral transfection of distinct types of human dendritic cells: high-efficiency gene transfer by electroporation into hematopoietic progenitor-but not monocyte-derived dendritic cells. Gene Ther 1998; 5: 700-707.
96. Lu L, Li W, Fu F, et al. Blockade of CD40-CD40 ligand pathway potentiates the capacity of donor-derived dendritic cell progenitors to induce long-term cardiac allograft survival. Transplantation 1997; 64: 1808-1815.
97. Takayama T, Tahara H, Thomson AW. Transduction of dendritic cell progenitors with a retroviral vector encoding viral interleukin-10 and enhanced green fluorescent protein allows purification of potentially tolerogenic antigen-presenting cells. Transplantation 1999; 68: 1903-1909.
98. Seroogy CM, Fathman CG. The application of gene therapy in autoimmune diseases. Gene Ther 2000; 7: 9-13.