Empirical advantages of adeno associated viral vectors for in vivo gene therapy for arthritis

Journal of Rheumatology

Volumn 27, Issue 4, 2000, Pages 983-989

  Goater, Jeffrey ^b   Müller, Rolf ^b   Kollias, George ^b   Firestein, Gary S ^b   Sanz, Iñaki ^b   O'Keefe, Regis J ^b   Schwarz, Edward M ^a  

^a UNIVERSITY OF ROCHESTER MEDICAL CENTER   (United States)

^b NONE

Author keywords

Adeno associated virus; Arthritis; Gene therapy; Inflammation; Transduction; Tumor necrosis factor

Indexed keywords

BETA GALACTOSIDASE; TUMOR NECROSIS FACTOR ALPHA; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTHRITIS; ARTICLE; CARTILAGE CELL; CHEMOLUMINESCENCE; CONTROLLED STUDY; DNA REPAIR; GAMMA IRRADIATION; GENE OVEREXPRESSION; GENE TARGETING; GENE THERAPY; GENETIC TRANSDUCTION; IMMUNOHISTOCHEMISTRY; MOUSE; NONHUMAN; PRIORITY JOURNAL; SYNOVIOCYTE; ULTRAVIOLET IRRADIATION;

ADENOVIRIDAE; ANIMALS; ARTHRITIS; CARTILAGE; CELLS, CULTURED; CYTOMEGALOVIRUS; FIBROBLASTS; GAMMA RAYS; GENE EXPRESSION REGULATION, VIRAL; GENE THERAPY; HELA CELLS; HUMANS; INTEGRINS; KNEE JOINT; LAC OPERON; MICE; MICE, INBRED CBA; MICE, TRANSGENIC; PROMOTER REGIONS (GENETICS); RECEPTORS, VITRONECTIN; SYNOVIAL MEMBRANE; TRANS-ACTIVATION (GENETICS); TRANSDUCTION, GENETIC; TRANSGENES; TUMOR NECROSIS FACTOR-ALPHA; ULTRAVIOLET RAYS;

EID: 0343618533     PISSN: 0315162X     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (50)

1. Feldmann M, Brennan FM, Maini RN. Rheumatoid arthritis. Cell 1996;85:307-10.
2. Yelin E. Arthritis. The cumulative impact of a common chronic condition. Arthritis Rheum 1992;35:489-97.
3. Harris ED. Clinical features of rheumatoid arthritis. In: W.N. Kelley, et al, editors. Textbook of rheumatology. 4th ed. New York: Saunders; 1993:874-911.
4. Evans CH, Robbins PD. Pathways to gene therapy in rheumatoid arthritis. Curr Opin Rheumatol 1996;8:230-4.
5. Evans CH, Ghivizzani SC, Kang R, et al. Gene therapy for rheumatic diseases. Arthritis Rheum 1999;42:1-16.
6. Verma IM, Somia N. Gene therapy - promises, problems and prospects [news]. Nature 1997;389:239-42.
7. Yovandich J, O'Malley BJ, Sikes M, Ledley FD. Gene transfer to synovial cells by intra-articular administration of plasmid DNA. Human Gene Therapy 1995;6:603-10.
8. 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 Therapy 1997;4:977-82.
9. 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 Therapy 1995;2:424-8.
10. Roessler BJ, Allen ED, Wilson JM, Hartman JW, Davidson BL. Adenoviral-mediated gene transfer to rabbit synovium in vivo. J Clin Invest 1993;92:1085-92.
11. Nita I, Ghivizzani SC, Galea-Lauri J, et al. Direct gene delivery to synovium. An evaluation of potential vectors in vitro and in vivo. Arthritis Rheum 1996;39:820-8.
12. Muzyczka N. Use of adeno-associated virus as a general transduction vector for mammalian cells [review]. Current Topics Microbiol Immunol 1992;158:97-129.
13. Berns KI. Parvovirus replication. Microbiol Rev 1990;54:316-29.
14. McLaughlin SK, Collis P, Hermonat PL, Muzyczka N. Adeno-associated virus general transduction vectors: analysis of proviral structures, J Virol 1988;62:1963-73.
15. Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 1989;63:3822-8.
16. Xiao X, Li J, Samulski RJ. Production of high-liter recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998;72:2224-32.
17. Russell DW, Miller AD, Alexander IE. Adeno-associated virus vectors preferentially transduce cells in S phase. Proc Nat Acad Sci USA 1994;91:8915-9.
18. Fisher KJ, Gao GP, Weitzman MD, DeMatteo R, Burda JF, Wilson JM. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 1996;70:520-32.
19. Koeberl DD, Alexander IE, Halbert CL, Russell DW, Miller AD. Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors. Proc Nat Acad Sci USA 1997;94:1426-31.
20. Snyder RO, Miao CH, Patijn GA, et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 1997;16:270-6.
21. Halbert CL, Standaert TA, Aitken ML, Alexander IE, Russell DW, Miller AD. Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration. J Virol 1997;71:5932-41.
22. Kaplitt MG, Leone P, Samulski RJ, et al. Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nat Genet 1994;8:148-54.
23. Fisher KJ, Jooss K, Alston J, et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nature Med 1997;3:306-12.
24. Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996;70:8098-108.
25. Samulski RJ, Zhu X, Xiao X, et al. Targeted integration of adeno-associated virus (AAV) into human chromosome 19 [published erratum appears in EMBO J 1992;11:1228]. EMBO J 1991; 10:3941-50.
26. Yakinoglu AO, Heilbronn R, Burkle A, Schlehofer J, Zur-Hausen H. DNA amplification of adeno-associated virus as a response to cellular genotoxic stress. Cancer Res 1988;48:3123-9.
27. Yakobson B, Koch T, Winocour E. Replication of adeno-associated virus in synchronized cells without the addition of a helper virus. J Virol 1987;61:972-81.
28. Alexander IE, Russell DW, Miller AD. DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J Virol 1994;68:8282-7.
29. Alexander IE, Russell DW, Spence AM, Miller AD. Effects of gamma irradiation on the trunsduction of dividing and nondividing cells in brain and muscle of rats by adeno-associated virus vectors. Human Gene Therapy 1996;7:841-50.
30. Smith CA, Farrah T, Goodwin RG. The TNF receptor superfamily of cellular and viral proteins: activation, costimulalion, and death. Cell 1994;76:959-63.
31. Feldmann M, Brennan FM, Maini RN. Role of cytokines in rheumatoid arthritis. Annu Rev Immunol 1996;14:397-440.
32. Douni E, Akassoglou K, Alexopoulou L, et al. Transgenic and knockout analyses of the role of TNF in immune regulation and disease pathogenesis. J Inflamm 1996;47:27-38.
33. Keffer J, Probert L, Cazlaris H, et al. Transgenic mice expressing human tumor necrosis factor: a predictive genetic model of arthritis. EMBO J 1991;10:4025-31.
34. Schwarz EM, Krimpenfort P, Berns A, Verma IM. Immunological defects in mice with a targeted disruption in Bcl-3. Genes Dev 1997;11:187-97.
35. Nguyen KH, Boyle DL, McCormack JE, Chada S, Jolly DJ, Firestein GS. Direct synovial gene transfer with retroviral vectors in rat adjuvant arthritis. J Rheumatol 1998;25:1118-25.
36. Firestein GS, Paine MM. Stromelysin and tissue inhibitor of metalloproteinases gene expression in rheumatoid arthritis synovium. Am J Pathol 1992;140:1309-14.
37. Schwarz EM, Badorff C, Hiura TS, et al. NpκB mediated inhibition of apoptosis is required for encephalomyocarditis virus virulence: a mechanism of resistance in p50 knockout mice. J Virol 1998; 72:5654-60.
38. Schwarz EM, Van Antwerp D, Verma IM. Constitutive phosphorylation of IκBα by casein kinase II occurs preferentially at serine 293: requirement for degradation of free IκBα. Mol Cell Biol 1996;16:3554-9.
39. Schwarz EM, Salgame P, Bloom BR. Molecular regulation of human interleukin 2 and T-cell function by interleukin 4. Proc Natl Acad Sci USA 1993;90:7734-8.
40. Summerford C, Bartlett JS. Samulski RJ, αVβ5 integrin: a co-receptor for adeno-associated virus type 2 infection. Nature Med 1999;5:78-82.
41. Ferrari FK, Samulski T, Shenk T, Samulski RJ. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J Virol 1996;70:3227-34.
42. 3H-thymidine incorporating cells in rheumatoid arthritis synovial membrane. Rheumatol Int 1986; 6:269-71.
43. Firestein GS, Yeo M, Zvaifler NJ. Apoptosis in rheumatoid arthritis synovium. J Clin Invest 1995;96:1631-8.
44. Nakajima T, Aono H, Hasunuma T, et al. Apoptosis and functional Fas antigen in rheumatoid arthritis synoviocytes. Arthritis Rheum 1995;38:485-91.
45. Mapp PI, Grootveld MC, Blake DR. Hypoxia, oxidative stress and rheumatoid arthritis [review]. Br Med Bull 1995;51:419-36.
46. Tak PP, Zvaifler NJ, Green DR, Firestein GS. Rheumatoid arthritis and p53: how oxidative stress might alter the course of inflammatory diseases. Immunol Today 2000;21:78-82.
47. Kaur H, Edmonds SE, Blake DR. Halliwell B. Hydroxyl radical generation by rheumatoid blood and knee joint synovial fluid. Ann Rheum Dis 1996;55:915-20.
48. 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;73:3410-7.
49. 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 antiarthritic effects. Proc Natl Acad Sci USA 1998;95:4613-8.
50. 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;162:3625-32.