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Therapeutic Gene Transfer for Rheumatoid Arthritis

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Book cover Gene Therapy of Autoimmune Diseases

Part of the book series: Medical Intelligence Unit ((MIUN))

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Conclusions

In RA, which is both a polyarticular and a systemic disease, anti-TNF or anti-IL-1 tar geted treatment will not be the only solutions proposed to patients in the future. Gene therapy approaches, when introduced, do not imply the exclusion of former therapies. They are only specific and promising examples of targeted therapies that are not based on the use of protein anymore, but rather on a gene encoding this protein. In the future, we will try to achieve a control of the gene expression, just as we tried in the past to control the protein administration in a biotherapy context (frequency of injection, dose of administered protein etc.). In the next few years, the challenges in the gene therapy of RA will be to improve the benefit-to-risk ratio of vector application, to achieve controlled gene expression, and to elucidate the immune consequences of transgene and vector administration. These parameters are essential and absolutely prerequired to pursue or enter upon any gene therapy clinical trials in patients suffering from rheumatoid arthritis.

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References

  1. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G et al. Gene therapy of human severe combined immunodeficiency (SCID)-Xl disease. Science 2000; 288:669–672.

    Article  PubMed  CAS  Google Scholar 

  2. 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; 348:255–256.

    Article  PubMed  Google Scholar 

  3. Kavanaugh AFLP. In: Gallin JI, Snyderman R, eds. Rheumatoid arthritis. 3rd ed. Lippincott Williams and Wilkins, 1999:1017–1037.

    Google Scholar 

  4. Elliott MJ, Maini RN, Feldmann M et al. Randomised double-blind comparison of chimeric monoclonal antibody to tumour necrosis factor alpha (cA2) versus placebo in rheumatoid arthritis. Lancet 1994; 344:1105–1110.

    Article  PubMed  CAS  Google Scholar 

  5. Moreland LW, Baumgartner SW, Schiff MH et al. Treatment of rheumatoid arthritis with a recombinant human tumor necrosis factor receptor (p75)-Fc fusion protein. N Engl J Med 1997; 337:141–147.

    Article  PubMed  CAS  Google Scholar 

  6. Campion GV, Lebsack ME, Lookabaugh J et al. Dose-range and dose-frequency study of recombinant human interleukin-1 receptor antagonist in patients with rheumatoid arthritis. The IL-1Ra Arthritis Study Group. Arthritis Rheum 1996; 39:1092–1101.

    Article  PubMed  CAS  Google Scholar 

  7. Bessis N, Doucet C, Cottard V et al. Gene therapy in rheumatoid arthritis. J Gene Med 2002; 4:581–591.

    Article  PubMed  CAS  Google Scholar 

  8. Ghivizzani SC, Oligino TJ, Glorioso JC et al. Gene therapy approaches for treating rheumatoid arthritis. Clin Orthop 2000; S288–299.

    Google Scholar 

  9. Fellowes R, Etheridge CJ, Coade S et al. Amelioration of established collagen induced arthritis by systemic IL-10 gene delivery. Gene Ther 2000; 7:967–977.

    Article  PubMed  CAS  Google Scholar 

  10. Bloquel C, Fabre E, Bureau MF et al. Plasmid DNA electrotransfer for intracellular and secreted proteins expression: New methodological developments and applications. J Gene Med 2004; (6Suppl 1):S11–23.

    Article  PubMed  CAS  Google Scholar 

  11. Saidenberg-Kermanach N, Bessis N, Deleuze V et al. Efficacy of interleukin-10 gene electrotransfer into skeletal muscle in mice with collagen-induced arthritis. J Gene Med 2003; 5:164–171.

    Article  CAS  Google Scholar 

  12. Bloquel C, Bessis N, Boissier MC et al. Gene therapy of collagen-induced arthritis by electrotransfer of human tumor necrosis factor-alpha soluble receptor I variants. Hum Gene Ther 2004; 15:189–201.

    Article  PubMed  CAS  Google Scholar 

  13. Kim JM, Ho SH, Hahn W et al. Electro-gene therapy of collagen-induced arthritis by using an expression plasmid for the soluble p75 tumor necrosis factor receptor-Fc fusion protein. Gene Ther 2003; 10:1216–1224.

    Article  PubMed  CAS  Google Scholar 

  14. Ho SH, Hahn W, Lee HJ et al. Protection against collagen-induced arthritis by electrotransfer of an expression plasmid for the interleukin-4. Biochem Biophys Res Commun 2004; 321:759–766.

    Article  PubMed  CAS  Google Scholar 

  15. Jeong JG, Kim JM, Ho SH et al. Electrotransfer of human IL-1Ra into skeletal muscles reduces the incidence of murine collagen-induced arthritis. J Gene Med 2004; 6:1125–1133.

    Article  PubMed  CAS  Google Scholar 

  16. Ghivizzani SC, Lechman ER, Tio C et al. Direct retrovirus-mediated gene transfer to the synovium of the rabbit knee: Implications for arthritis gene therapy. Gene Ther 1997; 4:977–982.

    Article  PubMed  CAS  Google Scholar 

  17. Makarov SS, Olsen JC, Johnston WN et al. Retrovirus mediated in vivo gene transfer to synovium in bacterial cell wall-induced arthritis in rats. Gene Ther 1995; 2:424–428.

    PubMed  CAS  Google Scholar 

  18. 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 USA 1996; 93:402–406.

    Article  PubMed  CAS  Google Scholar 

  19. Pap T, Gay RE, Gay S. Gene transfer: From concept to therapy. Curr Opin Rheumatol 2000; 12:205–210.

    Article  PubMed  CAS  Google Scholar 

  20. Rutkauskaite E, Zacharias W, Schedel J et al. Ribozymes that inhibit the production of matrix metalloproteinase 1 reduce the invasiveness of rheumatoid arthritis synovial fibroblasts. Arthritis Rheum 2004; 50:1448–1456.

    Article  PubMed  CAS  Google Scholar 

  21. Bandara G, Mueller GM, Galea-Lauri J et al. Intraarticular expression of biologically active interleukin 1-receptor-antagonist protein by ex vivo gene transfer. Proc Natl Acad Sci USA 1993; 90:10764–10768.

    Article  PubMed  CAS  Google Scholar 

  22. Evans CH, Ghivizzani SC, Smith P et al. Using gene therapy to protect and restore cartilage. Clin Orthop 2000; S214–219.

    Google Scholar 

  23. Gouze E, Ghivizzani SC, Palmer GD et al. Gene therapy for rheumatoid arthritis. Expert Opin Biol Ther 2001; 1:971–978.

    Article  PubMed  CAS  Google Scholar 

  24. Lin YL, Noel D, Mettling C et al. Feline immunodeficiency virus vectors for efficient transduction of primary human synoviocytes: Application to an original model of rheumatoid arthritis. Hum Gene Ther 2004; 15:588–596.

    Article  PubMed  CAS  Google Scholar 

  25. Wickham TJ. Targeting adenovirus. Gene Ther 2000; 7:110–114.

    Article  PubMed  CAS  Google Scholar 

  26. Otani K, Nita I, Macaulay W et al. Suppression of antigen-induced arthritis in rabbits by ex vivo gene therapy. J Immunol 1996; 156:3558–3562.

    PubMed  CAS  Google Scholar 

  27. Evans CH, Ghivizzani SC, Oligino TA et al. Future of adenoviruses in the gene therapy of arthritis. Arthritis Res 2001; 3:142–146.

    Article  PubMed  CAS  Google Scholar 

  28. Goossens PH, Schouten GJ, ’t Hart B et al. Feasibility of adenovirus-mediated nonsurgical synovectomy in collagen-induced arthritis-affected rhesus monkeys. Hum Gene Ther 1999; 10:1139–1149.

    Article  PubMed  CAS  Google Scholar 

  29. Sawchuk SJ, Boivin GP, Duwel LE et al. Anti-T cell receptor monoclonal antibody prolongs transgene expression following adenovirus-mediated in vivo gene transfer to mouse synovium. Hum Gene Ther 1996; 7:499–506.

    PubMed  CAS  Google Scholar 

  30. Jomary C, Vincent KA, Grist J et al. Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration. Gene Ther 1997; 4:683–690.

    Article  PubMed  CAS  Google Scholar 

  31. Haberman RP, McCown TJ, Samulski RJ. Inducible long-term gene expression in brain with adeno-associated virus gene transfer. Gene Ther 1998; 5:1604–1611.

    Article  PubMed  CAS  Google Scholar 

  32. Zhang L, Wang D, Fischer H et al. Efficient expression of CFTR function with adeno-associated virus vectors that carry shortened CFTR genes. Proc Natl Acad Sci USA 1998; 95:10158–10163.

    Article  PubMed  CAS  Google Scholar 

  33. Pan RY, Xiao X, Chen SL et al. Disease-inducible transgene expression from a recombinant adeno-associated virus vector in a rat arthritis model. J Virol 1999; 73:3410–3417.

    PubMed  CAS  Google Scholar 

  34. Goater J, Muller R, Kollias G et al. Empirical advantages of adeno associated viral vectors in vivo gene therapy for arthritis. J Rheumatol 2000; 27:983–989.

    PubMed  CAS  Google Scholar 

  35. Arai Y, Kubo T, Fushiki S et al. Gene delivery to human chondrocytes by an adeno associated virus vector. J Rheumatol 2000; 27:979–982.

    PubMed  CAS  Google Scholar 

  36. Cottard V, Mulleman D, Bouille P et al. Adeno-associated virus-mediated delivery of IL-4 prevents collagen-induced arthritis. Gene Ther 2000; 7:1930–1939.

    Article  PubMed  CAS  Google Scholar 

  37. Pan RY, Chen SL, Xiao X et al. Therapy and prevention of arthritis by recombinant adeno-associated virus vector with delivery of interleukin-1 receptor antagonist. Arthritis Rheum 2000; 43:289–297.

    Article  PubMed  CAS  Google Scholar 

  38. Zhang HG, Huang N, Liu D et al. Gene therapy that inhibits nuclear translocation of nuclear factor kappaB results in tumor necrosis factor alpha-induced apoptosis of human synovial fibroblasts. Arthritis Rheum 2000; 43:1094–1105.

    Article  PubMed  CAS  Google Scholar 

  39. Chan JM, Villarreal G, Jin W et al. Intraarticular gene transfer of TNFR:Fc suppresses experimental arthritis with reduced systemic distribution of the gene product. Mol Ther 2002; 6:727–736.

    Article  PubMed  CAS  Google Scholar 

  40. Bessis N, GarciaCozar FJ, Boissier MC. Immune responses to gene therapy vectors: Influence on vector function and effector mechanisms. Gene Ther 2004; 11(Suppl 1):S10–17.

    Article  PubMed  CAS  Google Scholar 

  41. Finck BK, Linsley PS, Wofsy D. Treatment of murine lupus with CTLA4Ig. Science 1994; 265:1225–1227.

    Article  PubMed  CAS  Google Scholar 

  42. Durie FH, Fava RA, Foy TM et al. Prevention of collagen-induced arthritis with an antibody to gp39, the ligand for CD40. Science 1993; 261:1328–1330.

    Article  PubMed  CAS  Google Scholar 

  43. Durie FH, Foy TM, Noelle RJ. The role of CD40 and its ligand (gp39) in peripheral and central tolerance and its contribution to autoimmune disease. Res Immunol 1994; 145:200–205, discussion 244–209.

    Article  PubMed  CAS  Google Scholar 

  44. Dayer JM. The saga of the discovery of IL-1 and TNF and their specific inhibitors in the pathogenesis and treatment of rheumatoid arthritis. Joint Bone Spine 2002; 69:123–132.

    Article  PubMed  Google Scholar 

  45. Boissier MC, Bessis N, Falgarone G. Options for blocking interleukin-1 in rheumatoid arthritis. Joint Bone Spine 2002; In press.

    Google Scholar 

  46. Colotta F, Re F, Muzio M et al. Interleukin-1 type II receptor: A decoy target for IL-1 that is regulated by IL-4. Science 1993; 261:472–475.

    Article  PubMed  CAS  Google Scholar 

  47. Burger D, Chicheportiche R, Giri JG et al. The inhibitory activity of human interleukin-1 receptor antagonist is enhanced by type II interleukin-1 soluble receptor and hindered by type I interleukin-1 soluble receptor. J Clin Invest 1995; 96:38–41.

    Article  PubMed  CAS  Google Scholar 

  48. Bessis N, Guery L, Mantovani A et al. The type II decoy receptor of IL-1 inhibits murine collagen-induced arthritis. Eur J Immunol 2000; 30:867–875.

    Article  PubMed  CAS  Google Scholar 

  49. 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.

    PubMed  CAS  Google Scholar 

  50. Le CH, Nicolson AG, Morales A et al. Suppression of collagen-induced arthritis through adenovirus-mediated transfer of a modified tumor necrosis factor alpha receptor gene. Arthritis Rheum 1997; 40:1662–1669.

    Article  PubMed  CAS  Google Scholar 

  51. Kim SH, Evans CH, Kim S et al. Gene therapy for established murine collagen-induced arthritis by local and systemic adenovirus-mediated delivery of interleukin-4. Arthritis Res 2000; 2:293–302.

    Article  PubMed  CAS  Google Scholar 

  52. Oligino T, Ghivizzani S, Wolfe D et al. Intra-articular delivery of a herpes simplex virus IL-1Ra gene vector reduces inflammation in a rabbit model of arthritis. Gene Ther 1999; 6:1713–1720.

    Article  PubMed  CAS  Google Scholar 

  53. Chernajovsky Y, Adams G, Podhajcer OL et al. Inhibition of transfer of collagen-induced arthritis into SCID mice by ex vivo infection of spleen cells with retroviruses expressing soluble tumor necrosis factor receptor. Gene Ther 1995; 2:731–735.

    PubMed  CAS  Google Scholar 

  54. Bessis N, Boissier MC, Ferrara P et al. Attenuation of collagen-induced arthritis in mice by treatment with vector cells engineered to secrete interleukin-13. Eur J Immunol 1996; 26:2399–2403.

    PubMed  CAS  Google Scholar 

  55. Bessis N, Chiocchia G, Kollias G et al. Modulation of proinflammatory cytokine production in tumour necrosis factor-alpha (TNF-alpha)-transgenic mice by treatment with cells engineered to secrete IL-4, IL-10 or IL-13. Clin Exp Immunol 1998; 111:391–396.

    Article  PubMed  CAS  Google Scholar 

  56. 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 (CIA). Clin Exp Immunol 1999; 117:376–382.

    Article  PubMed  CAS  Google Scholar 

  57. Apparailly F, Verwaerde C, Jacquet C et al. Adenovirus mediated transfer of viral IL-10 gene inhibits collagen-induced arthritis. J Immunol 1998; 160:5213–5220.

    PubMed  CAS  Google Scholar 

  58. Lechman ER, Jaffurs D, Ghivizzani SC et al. Direct adenoviral gene transfer of viral IL-10 to rabbit knees with experimental arthritis ameliorates disease in both injected and contralateral control knees. J Immunol 1999; 163:2202–2208.

    PubMed  CAS  Google Scholar 

  59. 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.

    PubMed  CAS  Google Scholar 

  60. Trippel SB. Growth factor actions on articular cartilage. J Rheumatol Suppl 1995; 43:129–132.

    PubMed  CAS  Google Scholar 

  61. Okamoto K, Asahara H, Kobayashi T et al. Induction of apoptosis in the rheumatoid synovium by Fas ligand gene transfer. Gene Ther 1998; 5:331–338.

    Article  PubMed  CAS  Google Scholar 

  62. Zhang H, Yang Y, Horton JL et al. Amelioration of collagen-induced arthritis by CD95 (Apo-1/Fas)-ligand gene transfer. J Clin Invest 1997; 100:1951–1957.

    PubMed  CAS  Google Scholar 

  63. Kobayashi T, Okamoto K, Kobata T et al. Novel gene therapy for rheumatoid arthritis by FADD gene transfer: Induction of apoptosis of rheumatoid synoviocytes but not chondrocytes. Gene Ther 2000; 7:527–533.

    Article  PubMed  CAS  Google Scholar 

  64. Kim SH, Kim S, Evans CH et al. Effective treatment of established murine collagen-induced arthritis by systemic administration of dendritic cells genetically modified to express IL-4. J Immunol 2001; 166:3499–3505.

    PubMed  CAS  Google Scholar 

  65. Chernajovsky Y, Adams G, Triantaphyllopoulos K et al. Pathogenic lymphoid cells engineered to express TGF beta 1 ameliorate disease in a collagen-induced arthritis model. Gene Ther 1997; 4:553–559.

    Article  PubMed  CAS  Google Scholar 

  66. Bessis N, Cottard V, Saidenberg-Kermanach’ N et al. Syngeneic fibroblasts transfected with a plasmid encoding interleukin-4 as nonviral vectors for anti-inflammatory gene therapy in collagen-induced arthritis. J Gene Med 2002; 4:300–307.

    Article  PubMed  Google Scholar 

  67. 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 protein in the knee. Arthritis Rheum 1997; 40:893–900.

    Article  PubMed  CAS  Google Scholar 

  68. Evans CH, Robbins PD, Ghivizzani SC et al. Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially anti-arthritic cytokine gene to human joints with rheumatoid arthritis. Hum Gene Ther 1996; 7:1261–1280.

    PubMed  CAS  Google Scholar 

  69. Baragi VM. MFG-IRAP University of pittsburgh. Curr Opin Investig Drugs 2000; 1:194–198.

    PubMed  CAS  Google Scholar 

  70. Taniguchi K, Kohsaka H, Inoue N et al. Induction of the p16INK4a senescence gene as a new therapeutic strategy for the treatment of rheumatoid arthritis. Nat Med 1999; 5:760–767.

    Article  PubMed  CAS  Google Scholar 

  71. Bakker A, van de Loo FAJ, Bennink M et al. Inflammation-inducible intra-articular production of human IL-1 receptor antagonist results in a more efficient inhibition of collagen-induced arthritis than does constitutive expression of the same transgene. Arthritis res 2001; 3(suppl 1):A2.

    Article  Google Scholar 

  72. 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 USA 1998; 95:4613–4618.

    Article  PubMed  CAS  Google Scholar 

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Authors and Affiliations

  1. UPRES EA-3408, Immunology, Université Paris 13, 74 rue Marcel Cachin, Bobigny cedex, France

    Natacha Bessis & Marie-Christophe Boissier

  2. Service de Rhumatologie, Avicenne Hospital, AP-HP, Bobigny, France

    Marie-Christophe Boissier

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  1. Natacha Bessis
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Bessis, N., Boissier, MC. (2005). Therapeutic Gene Transfer for Rheumatoid Arthritis. In: Gene Therapy of Autoimmune Diseases. Medical Intelligence Unit. Springer, Boston, MA. https://doi.org/10.1007/0-387-28670-5_5

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