RNA interference-based gene therapy for successful treatment of rheumatoid arthritis

Expert Opinion on Biological Therapy

Volumn 9, Issue 5, 2009, Pages 535-538

  Courties, Gabriel ^a,b   Presumey, Jessy ^a,b   Duroux Richard, Isabelle ^a,b   Jorgensen, Christian ^a,b,c   Apparailly, Florence ^a,b,c  

^a INSERM   (France)

^b UNIV MONTPELLIER   (France)

^c LAPEYRONIE HOSPITAL   (France)

Author keywords

Gene therapy; Inflammation; Rheumatoid arthritis; RNA interference

Indexed keywords

ADENOVIRUS VECTOR; CHITOSAN; DIOLEOYLPHOSPHATIDYLETHANOLAMINE; INTERLEUKIN 18; INTERLEUKIN 1BETA; INTERLEUKIN 6; LENTIVIRUS VECTOR; LIPOPLEX; LIPOSOME; NANOPARTICLE; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; TUMOR NECROSIS FACTOR ANTIBODY;

CLINICAL TRIAL; DRUG EFFICACY; GENE EXPRESSION; GENE SILENCING; GENE THERAPY; HUMAN; NONHUMAN; NONVIRAL GENE THERAPY; REVIEW; RHEUMATOID ARTHRITIS; RNA ANALYSIS; RNA INTERFERENCE; VIRAL GENE THERAPY;

ANIMALS; ARTHRITIS, RHEUMATOID; GENE THERAPY; HUMANS; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 67649094527     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712590902926089     Document Type: Review

References (29)

1. Jinek M, Doudna JA. A three-dimensional view of the molecular machinery of RNA interference. Nature 2009;457(7228):405-412
2. Pillai RS, Bhattacharyya SN, Filipowicz W. Repression of protein synthesis by miRNAs: how many mechanisms? Trends Cell Biol 2007;17(3):118-126
3. de Fougerolles A, Vornlocher HP, Maraganore J, Lieberman J. Interfering with disease: a progress report on siRNA-based therapeutics. Nat Rev Drug Discov 2007;6(6):443-453 (Pubitemid 47064567)
4. Song E, Lee SK, Wang J, et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med 2003;9(3):347-351 (Pubitemid 36362792)
5. Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference- based therapeutics. Nature 2009;457(7228):426-433
6. Kinne RW, Stuhlmuller B, Burmester GR. Cells of the synovium in rheumatoid arthritis. Macrophages. Arthritis Res Ther 2007;9(6):224. Published online 21 December 2007, doi:10.1186/ar2333
7. McInnes IB, Schett G. Cytokines in the pathogenesis of rheumatoid arthritis. Nat Rev Immunol 2007;7(6):429-442 (Pubitemid 46834849)
8. Bongartz T, Sutton AJ, Sweeting MJ, et al. Anti-TNF antibody therapy in rheumatoid arthritis and the risk of serious infections and malignancies: systematic review and meta-analysis of rare harmful effects in randomized controlled trials. JAMA 2006;295(19):2275-2285 (Pubitemid 43736578)
9. Gabriel SE. Tumor necrosis factor inhibition: a part of the solution or a part of the problem of heart failure in rheumatoid arthritis? Arthritis Rheum 2008;58(3):637-640
10. Filleur S, Courtin A, Ait-Si-Ali S, et al. siRNA-mediated inhibition of vascular endothelial growth factor severely limits tumor resistance to antiangiogenic thrombospondin-1 and slows tumor vascularization and growth. Cancer Res 2003;63(14):3919-3922 (Pubitemid 36917906)
11. Tolentino MJ, Brucker AJ, Fosnot J, et al. Intravitreal injection of vascular endothelial growth factor small interfering RNA inhibits growth and leakage in a nonhuman primate, laser-induced model of choroidal neovascularization. Retina 2004;24(1):132-138 (Pubitemid 38240851)
12. Corey DR. Chemical modification: the key to clinical application of RNA interference? J Clin Invest 2007;117(12):3615-3622
13. Akhtar S, Benter IF. Nonviral delivery of synthetic siRNAs in vivo. J Clin Invest 2007;117(12):3623-3632 (Pubitemid 350224068)
14. Whitehead KA, Langer R, Anderson DG. Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov 2009;8(2):129-138
15. Grimm D, Streetz KL, Jopling CL, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006;441(7092):537-541 (Pubitemid 44050158)
16. Khoury M, Louis-Plence P, Escriou V, et al. Efficient new cationic liposome formulation for systemic delivery of small interfering RNA silencing tumor necrosis factor α in experimental arthritis. Arthritis Rheum 2006;54(6):1867-1877 (Pubitemid 43877936)
17. Howard KA, Paludan SR, Behlke MA, et al. Chitosan/siRNA nanoparticle-mediated TNF-α knockdown in peritoneal macrophages for anti-inflammatory treatment in a murine arthritis model. Mol Ther 2009;17(1):162-168
18. Brennan FM, McInnes IB. Evidence that cytokines play a role in rheumatoid arthritis. J Clin Invest 2008;118(11):3537-3545
19. Khoury M, Escriou V, Courties G, et al. Combined anti-cytokine siRNA lipoplexes efficiently suppress arthritis. Arthritis Rheum 2008;58(8):2356-2367
20. Inoue A, Takahashi KA, Mazda O, et al. Electro-transfer of small interfering RNA ameliorated arthritis in rats. Biochem Biophys Res Commun 2005;336(3):903-908 (Pubitemid 41338354)
21. Schiffelers RM, Xu J, Storm G, et al. Effects of treatment with small interfering RNA on joint inflammation in mice with collagen-induced arthritis. Arthritis Rheum 2005;52(4):1314-1318 (Pubitemid 40530142)
22. Lai Kwan Lam Q, King Hung Ko O, Zheng BJ, Lu L. Local BAFF gene silencing suppresses Th17-cell generation and ameliorates autoimmune arthritis. Proc Natl Acad Sci U S A 2008;105(39):14993-14998
23. Williams DA. RAC reviews serious adverse event associated with AAV therapy trial. Mol Ther 2007;15(12):2053-2054 (Pubitemid 350148916)
24. Apparailly F, Khoury M, Vervoordeldonk MJ, et al. Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints. Hum Gene Ther 2005;16(4):426-434 (Pubitemid 40647140)
25. Adriaansen J, Tas SW, Klarenbeek PL, et al. Enhanced gene transfer to arthritic joints using adeno-associated virus type 5: implications for intra-articular gene therapy. Ann Rheum Dis 2005;64(12):1677-1684 (Pubitemid 41667139)
26. Randolph GJ, Jakubzick C, Qu C. Antigen presentation by monocytes and monocyte-derived cells. Curr Opin Immunol 2008;20(1):52-60
27. Evans HG, Suddason T, Jackson I, et al. Optimal induction of T helper 17 cells in humans requires T cell receptor ligation in the context of Toll-like receptor-activated monocytes. Proc Natl Acad Sci U S A 2007;104(43):17034-17039 (Pubitemid 350210986)
28. Valaperti A, Marty RR, Kania G, et al. CD11b+ monocytes abrogate Th17 CD4+ T cell-mediated experimental autoimmune myocarditis. J Immunol 2008;180(4):2686-2695
29. Khoury M, Jorgensen C, Apparailly F. RNAi in arthritis: prospects of a future antisense therapy in inflammation. Curr Opin Mol Ther 2007;9(5):483-489 (Pubitemid 47577405)