Technology insight: Gene transfer and the design of novel treatments for rheumatoid arthritis

Nature Clinical Practice Rheumatology

Volumn 2, Issue 3, 2006, Pages 153-162

  Moritz, Falk ^a   Distler, Oliver ^a   Ospelt, Caroline ^a   Gay, Renate E ^a   Gay, Steffen ^a,b  

^a UNIVERSITY OF ZURICH   (Switzerland)

^b WORLD HEALTH ORGANIZATION   (Switzerland)

Author keywords

Gene transfer; Inflammation; Matrix degradation; Rheumatoid arthritis; Synovial fibroblasts

Indexed keywords

ANGIOPOIETIN RECEPTOR; ANGIOSTATIN; ENDOSTATIN; FAS ASSOCIATED DEATH DOMAIN PROTEIN; FAS LIGAND; IMMUNOGLOBULIN ENHANCER BINDING PROTEIN; INTERLEUKIN 1 RECEPTOR; INTERLEUKIN 10; INTERLEUKIN 13; INTERLEUKIN 17; INTERLEUKIN 18; INTERLEUKIN 4; INTERSTITIAL COLLAGENASE; LENTIVIRUS VECTOR; MYC PROTEIN; PARVOVIRUS VECTOR; PLASMID VECTOR; PLASMINOGEN ACTIVATOR INHIBITOR; PROTEIN P16; PROTEIN P21; PROTEIN P53; RAF PROTEIN; RAS PROTEIN; RETROVIRUS VECTOR; SUPPRESSOR OF CYTOKINE SIGNALING 3; THROMBOSPONDIN 1; THYMIDINE KINASE; TISSUE INHIBITOR OF METALLOPROTEINASE 1; TISSUE INHIBITOR OF METALLOPROTEINASE 3; TRANSFORMING GROWTH FACTOR BETA1; TUMOR NECROSIS FACTOR RECEPTOR; TUMOR NECROSIS FACTOR RELATED APOPTOSIS INDUCING LIGAND; UNINDEXED DRUG; VASCULOTROPIN RECEPTOR 1;

BIOTECHNOLOGY; BONE DESTRUCTION; CARTILAGE DEGENERATION; CLINICAL PROTOCOL; DENDRITIC CELL; DRUG MECHANISM; DRUG TARGETING; FEASIBILITY STUDY; GENE EXPRESSION REGULATION; GENE TARGETING; GENE TRANSFER; HUMAN; IN VITRO STUDY; IN VIVO STUDY; MEDICAL RESEARCH; NONVIRAL GENE DELIVERY SYSTEM; PATHOGENESIS; PRIORITY JOURNAL; REVIEW; RHEUMATOID ARTHRITIS; VIRAL GENE DELIVERY SYSTEM;

ANIMALS; ARTHRITIS, RHEUMATOID; DISEASE MODELS, ANIMAL; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; MICE; MICE, SCID; RHEUMATOLOGY;

EID: 33646836119     PISSN: 17458382     EISSN: 17458390     Source Type: Journal    
DOI: 10.1038/ncprheum0117     Document Type: Review

References (67)

1. Pierer M et al. (2003) The SCID mouse model: Novel therapeutic targets - lessons from gene transfer. Springer Semin Immunopathol 25: 65-78
2. van de Loo FA et al. (2004) Gene therapy in animal models of rheumatoid arthritis: Are we ready for the patients? Arthritis Res Ther 6: 183-196
3. Tarner IH et al. (2003) Treatment of autoimmune disease by adoptive cellular gene therapy. Ann NY Acad Sci 998: 512-519
4. Annenkov A and Chernajovsky Y (2000) Engineering mouse T lymphocytes specific to type II collagen by transduction with a chimeric receptor consisting of a single chain Fv and TCR zeta. Gene Ther 7: 714-722
5. Hacein-Bey-Abina S et al. (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419
6. Distler JH et al. (2005) Nucleofection: A new, highly efficient transfection method for primary human keratinocytes. Exp Dermatol 14: 315-320
7. Adriaansen J et al. (2006) A novel approach for gene therapy: engraftment of fibroblasts containing the artificial chromosome expression system at the site of inflammation. J Gene Med 8: 63-71
8. Basu J and Willard HF (2005) Artificial and engineered chromosomes: non-integrating vectors for gene therapy. Trends Mol Med 11: 251-258
9. Goossens PH et al. (2001) The influence of synovial fluid on adenovirus-mediated gene transfer to the synovial tissue. Arthritis Rheum 44: 48-52
10. Apparailly F et al. (2005) Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints. Hum Gene Ther 16: 426-434
11. Gould DJ et al. (2004) Inhibition of established collagen-induced arthritis with a tumour necrosis factor-α inhibitor expressed from a self-contained doxycycline regulated plasmid. Arthritis Res Ther 6: R103-R113
12. Ohashi S et al. (2002) Successful genetic transduction in vivo into synovium by means of electroporation. Biochem Biophys Res Commun 293: 1530-1535
13. Evans CH (2005) Gene therapy: What have we accomplished and where do we go from here? J Rheumatol Suppl 72: 17-20
14. Pan RY et al. (1999) Disease-inducible transgene expression from a recombinant adeno-associated virus vector in a rat arthritis model. J Virol 73: 3410-3417
15. Gouze E et al. (2003) Lentiviral-mediated gene delivery to synovium: potent intra-articular expression with amplification by inflammation. Mol Ther 7: 460-466
16. Corbel SY and Rossi FM (2002) Latest developments and in vivo use of the Tet system: Ex vivo and in vivo delivery of tetracycline-regulated genes. Curr Opin Biotechnol 13: 448-452
17. Wieser C et al. (2005) Regulated and constitutive expression of anti-inflammatory cytokines by nontransforming herpesvirus saimiri vectors. Gene Ther 12: 395-406
18. Varley AW et al. (1997) A two-component expression system that responds to inflammatory stimuli in vivo. Nat Biotechnol 15: 1002-1006
19. Miagkov AV et al. (2002) Endogenous regulation of a therapeutic transgene restores homeostasis in arthritic joints. J Clin Invest 109: 1223-1229
20. Bakker AC et al. (2002) C3-Tat/HIV-regulated intraarticular human interleukin-1 receptor antagonist gene therapy results in efficient inhibition of collagen-induced arthritis superior to cytomegalovirus-regulated expression of the same transgene. Arthritis Rheum 46: 1661-1670
21. Pugliese A et al. (2005) A review of HIV-1 Tat protein biological effects. Cell Biochem Funct 23: 223-227
22. van de Loo FA et al. (2004) An inflammation-inducible adenoviral expression system for local treatment of the arthritic joint. Gene Ther 11: 581-590
23. Kim SH et al. (2002) Ex vivo gene delivery of IL-1Ra and soluble TNF receptor confers a distal synergistic therapeutic effect in antigen-induced arthritis. Mol Ther 6: 591-600
24. Müller-Ladner U et al. (1997) Human IL-1Ra gene transfer into human synovial fibroblasts is chondroprotective. J Immunol 158: 3492-3498
25. Ghivizzani SC et al. (1998) Direct adenovirus-mediated gene transfer of interleukin 1 and tumor necrosis factor α soluble receptors to rabbit knees with experimental arthritis has local and distal anti-arthritic effects. Proc Natl Acad Sci USA 95: 4613-4618
26. Evans CH et al. (2005) Gene transfer to human joints: Progress toward a gene therapy of arthritis. Proc Natl Acad Sci USA 102: 8698-8703
27. Bresnihan B (2002) Anakinra as a new therapeutic option in rheumatoid arthritis: Clinical results and perspectives. Clin Exp Rheumatol 20: S32-S34
28. Zhang HG et al. (2000) Adeno-associated virus production of soluble tumor necrosis factor receptor neutralizes tumor necrosis factor alpha and reduces arthritis. Hum Gene Ther 11: 2431-2442
29. Müller-Ladner U et al. (1999) Gene transfer of cytokine inhibitors into human synovial fibroblasts in the SCID mouse model. Arthritis Rheum 42: 490-497
30. Quattrocchi E et al. (1999) Paradoxical effects of adenovirus-mediated blockade of TNF activity in murine collagen-induced arthritis. J Immunol 163: 1000-1009
31. Bloquel C et al. (2004) Gene therapy of collagen-induced arthritis by electrotransfer of human tumor necrosis factor-α soluble receptor I variants. Hum Gene Ther 15: 189-201
32. Mease P et al. A phase 1 dose-escalation study of intra-articular administration of a recombinant adeno-associated vector containing the TNFR:FC fusion gene, in inflammatory arthritis. 13th Annual Congress of the European Society of Gene Therapy 2005, Oct 29-Nov 1
33. Lubberts E et al. (2000) Intra-articular IL-10 gene transfer regulates the expression of collagen-induced arthritis (CIA) in the knee and ipsilateral paw. Clin Exp Immunol 120: 375-383
34. Neidhart M et al. (2004) Deficient expression of interleukin-10 receptor α-chain in rheumatoid arthritis synovium - limitation of inflammatory animal models. Arthritis Rheum 50: S112
35. Cottard V et al. (2000) Adeno-associated virus-mediated delivery of IL-4 prevents collagen-induced arthritis. Gene Ther 7: 1930-1939
36. Woods JM et al. (2001) IL-4 adenoviral gene therapy reduces inflammation, proinflammatory cytokines, vascularization, and bony destruction in rat adjuvant-induced arthritis. J Immunol 166: 1214-1222
37. Nabbe KC et al. (2005) Local IL-13 gene transfer prior to immune-complex arthritis inhibits chondrocyte death and matrix-metalloproteinase-mediated cartilage matrix degradation despite enhanced joint inflammation. Arthritis Res Ther 7: R392-R401
38. Koenders MI et al. (2005) Induction of cartilage damage by overexpression of T cell interleukin-17A in experimental arthritis in mice deficient in interleukin-1. Arthritis Rheum 52: 975-983
39. Joosten LA et al. (2004) Interleukin-18 promotes joint inflammation and induces interleukin-1-driven cartilage destruction. Am J Pathol 165: 959-967
40. Song XY et al. (1998) Plasmid DNA encoding transforming growth factor-β1 suppresses chronic disease in a streptococcal cell wall-induced arthritis model. J Clin Invest 101: 2615-2621
41. Tak PP et al. (2001) Inhibitor of nuclear factor κB kinase β is a key regulator of synovial inflammation. Arthritis Rheum 44: 1897-1907
42. Miagkov AV et al. (1998) NF-κB activation provides the potential link between inflammation and hyperplasia in the arthritic joint. Proc Natl Acad Sci USA 95: 13859-13864
43. Yamamoto A et al. (2003) Suppression of arthritic bone destruction by adenovirus-mediated dominant-negative Ras gene transfer to synoviocytes and osteoclasts. Arthritis Rheum 48: 2682-2692
44. Pap T et al. (2004) Cooperation of Ras- and c-Myc-dependent pathways in regulating the growth and invasiveness of synovial fibroblasts in rheumatoid arthritis. Arthritis Rheum 50: 2794-2802
45. Wakisaka S et al. (1998) Possible correction of abnormal rheumatoid arthritis synovial cell function by jun D transfection in vitro. Arthritis Rheum 41: 470-481
46. Krause A et al. (2002) Rheumatoid arthritis synoviocyte survival is dependent on Stat3. J Immunol 169: 6610-6616
47. Shouda T et al. (2001) Induction of the cytokine signal regulator SOCS3/ CIS3 as a therapeutic strategy for treating inflammatory arthritis. J Clin Invest 108: 1781-1788
48. Sherr CJ and Roberts JM (1999) CDK inhibitors: Positive and negative regulators of G1-phase progression. Genes Dev 13: 1501-1512
49. Yao Q et al. (2001) Gene transfer of p53 to arthritic joints stimulates synovial apoptosis and inhibits inflammation. Mol Ther 3: 901-910
50. Nasu K et al. (2000) Adenoviral transfer of cyclin-dependent kinase inhibitor genes suppresses collagen-induced arthritis in mice. J Immunol 165: 7246-7252
51. Yao Q et al. (2003) Intra-articular adenoviral-mediated gene transfer of trail induces apoptosis of arthritic rabbit synovium. Gene Ther 10: 1055-1060
52. Yao Q et al. (2000) Adenoviral mediated delivery of FAS ligand to arthritic joints causes extensive apoptosis in the synovial lining. J Gene Med 2: 210-219
53. Kobayashi T et al. (2000) Novel gene therapy for rheumatoid arthritis by FADD gene transfer: Induction of apoptosis of rheumatoid synoviocytes but not chondrocytes. Gene Ther 7: 527-533
54. Sant SM et al. (1998) Molecular lysis of synovial lining cells by in vivo herpes simplex virus-thymidine kinase gene transfer. Hum Gene Ther 9: 2735-2743
55. Goossens PH et al. (1999) Feasibility of adenovirus-mediated nonsurgical synovectomy in collagen-induced arthritis-affected rhesus monkeys. Hum Gene Ther 10: 1139-1149
56. Distler JH et al. (2003) Angiogenic and angiostatic factors in the molecular control of angiogenesis. Q J Nucl Med 47: 149-161
57. Afuwape AO et al. (2003) Adenoviral delivery of soluble VEGF receptor 1 (sFlt-1) abrogates disease activity in murine collagen-induced arthritis. Gene Ther 10: 1950-1960
58. Chen Y et al. (2005) Gene therapy targeting the Tie2 function ameliorates collagen-induced arthritis and protects against bone destruction. Arthritis Rheum 52: 1585-1594
59. Yin G et al. (2002) Endostatin gene transfer inhibits joint angiogenesis and pannus formation in inflammatory arthritis. Mol Ther 5: 547-554
60. Kim JM et al. (2002) Angiostatin gene transfer as an effective treatment strategy in murine collagen-induced arthritis. Arthritis Rheum 46: 793-801
61. Jou IM et al. (2005) Thrombospondin 1 as an effective gene therapeutic strategy in collagen-induced arthritis. Arthritis Rheum 52: 339-344
62. Rutkauskaite E et al. (2004) Ribozymes that inhibit the production of matrix metalloproteinase 1 reduce the invasiveness of rheumatoid arthritis synovial fibroblasts. Arthritis Rheum 50: 1448-1456
63. van der Laan WH et al. (2003) Cartilage degradation and invasion by rheumatoid synovial fibroblasts is inhibited by gene transfer of TIMP-1 and TIMP-3. Gene Ther 10: 234-242
64. Apparailly F et al. (2002) Adenovirus-mediated gene transfer of urokinase plasminogen inhibitor inhibits angiogenesis in experimental arthritis. Gene Ther 9: 192-200
65. van der Laan WH et al. (2000) Cartilage degradation and invasion by rheumatoid synovial fibroblasts is inhibited by gene transfer of a cell surface-targeted plasmin inhibitor. Arthritis Rheum 43: 1710-1718
66. Schedel J et al. (2004) Targeting cathepsin L (CL) by specific ribozymes decreases CL protein synthesis and cartilage destruction in rheumatoid arthritis. Gene Ther 11: 1040-1047
67. Saidenberg-Kermanac'h N et al. (2003) Efficacy of interleukin-10 gene electrotransfer into skeletal muscle in mice with collagen-induced arthritis. J Gene Med 5: 164-171