Getting arthritis gene therapy into the clinic

Nature Reviews Rheumatology

Volumn 7, Issue 4, 2011, Pages 244-249

  Evans, Christopher H ^a   Ghivizzani, Steven C ^b   Robbins, Paul D ^c  

^a HARVARD MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

^c UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; COMPLEMENTARY DNA; ETANERCEPT; GALSULFASE; INTERLEUKIN 1 RECEPTOR BLOCKING AGENT; TRANSFORMING GROWTH FACTOR BETA1; VIRUS VECTOR;

ANKYLOSING SPONDYLITIS; ARTHRITIS; DRUG INDICATION; DRUG SAFETY; EX VIVO GENE TRANSFER; FUNDING; GENE INSERTION; GENE THERAPY; HEMOPHILIA; HUMAN; IN VIVO GENE TRANSFER; MAROTEAUX LAMY SYNDROME; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; OSTEOARTHRITIS; PATIENT SAFETY; PIGMENTED VILLONODULAR SYNOVITIS; PRIORITY JOURNAL; PSORIATIC ARTHRITIS; REVIEW; RHEUMATOID ARTHRITIS; SYNOVECTOMY; TRANSGENE; VIRUS GENE;

ARTHRITIS, RHEUMATOID; CLINICAL TRIALS AS TOPIC; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; OSTEOARTHRITIS; RHEUMATOLOGY;

EID: 79953662850     PISSN: 17594790     EISSN: 17594804     Source Type: Journal    
DOI: 10.1038/nrrheum.2010.193     Document Type: Review

References (32)

1. Bandara, G. et al. Gene transfer to synoviocytes: prospects for gene treatment of arthritis. DNA Cell Biol. 11, 227-231 (1992).
2. Mease, P J. et al. Safety, tolerability, and clinical outcomes after intraarticular injection of a recombinant adeno-associated vector containing a tumor necrosis factor antagonist gene: results of a phase 1/2 study. J. Rheumatol. 37, 692-703 (2010).
3. Traister, R. S. & Hirsch, R. Gene therapy for arthritis. Mod. Rheumatol. 18, 2-14 (2008).
4. Ghivizzani, S. C et al. Perspectives on the use of gene therapy for chronic joint diseases. Curr. Gene Ther. 8, 273-286 (2008).
5. Gouze, E. et al. Transgene persistence and cel turnover in the diarthrodial joint: implications for gene therapy of chronic joint diseases. Mol. Ther. 15, 1114-1120 (2007). (Pubitemid 46813614)
6. Madry, H., Cucchiarini, M., Terwilliger, E. F. & Trippel, S. B. Recombinant adeno-associated virus vectors efficiently and persistently transduce chondrocytes in normal and osteoarthritic human articular cartilage. Hum. Gene Ther. 14, 393-402 (2003). (Pubitemid 36308358)
7. Zhang, H., Gao, G., Clayburne, G. & Schumacher, H. R. Elimination of rheumatoid synovium in situ using a Fas ligand 'gene scalpel'. Arthritis Res. Ther. 7, R1235-R1243 (2005).
8. Goossens, P. H. et al. Feasibility of adenovirus-mediated nonsurgical synovectomy in collagen-induced arthritis-affected rhesus monkeys. Hum. Gene Ther. 10, 1139-1149 (1999). (Pubitemid 29231858)
9. Sant, S. M. et al. Molecular lysis of synovial ining cells by in vivo herpes simplex virus-thymidine kinase gene transfer. Hum. Gene Ther. 9, 2735-2743 (1998). (Pubitemid 29027017)
10. Miagkov, A. V., Varley, A. W., Munford, R. S. & Makarov, S. S. Endogenous regulation of a therapeutic transgene restores homeostasis in arthritic joints. J. Clin. Invest. 109, 1223-1229 (2002). (Pubitemid 34496600)
11. Evans, C H. et al. Gene transfer to human joints: progress toward a gene therapy of arthritis. Proc Natl Acad. Sci. USA 102, 8698-8703 (2005). (Pubitemid 40862798)
12. Bendele, A. et al. Efficacy of sustained blood levels of interleukin-1 receptor antagonist in animal models of arthritis: comparison of efficacy in animal models with human clinical data. Arthritis Rheum. 42, 498-506 (1999). (Pubitemid 29136098)
13. Makarov, S. S. et al. Suppression of experimental arthritis by gene transfer of interleukin 1 receptor antagonist cDNA. Proc Natl Acad. Sci. USA 93, 402-406 (1996). (Pubitemid 26038167)
14. Bandara, G. et al. Intraarticular expression of biologically active interleukin 1-receptor-antagonist protein by ex vivo gene transfer. Proc Natl Acad. Sci. USA 90, 10764-10768 (1993). (Pubitemid 23347123)
15. Wehling, P et al. Clinical responses to gene therapy in joints of two subjects with rheumatoid arthritis. Hum. Gene Ther. 20, 97-101 (2009).
16. Kohn, D. B., Sadelain, M. & Glorioso, J. C Occurrence of leukaemia following gene therapy of X-linked SCID. Nat. Rev. Cancer 3, 477-488 (2003). (Pubitemid 37328836)
17. Miles, B. J. et al. Prostate-specific antigen response and systemic T cell activation after in situ gene therapy in prostate cancer patients failing radiotherapy. Hum. Gene Ther. 12, 1955-1967 (2001). (Pubitemid 33116762)
18. Raper, S. E. et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol. Genet. Metab. 80, 148-158 (2003). (Pubitemid 37272241)
19. Mease, P J. et al. Local delivery of a recombinant adenoassociated vector containing a tumor necrosis factor alpha antagonist gene in inflammatory arthritis: a phase 1 dose-escalation safety and tolerability study. Ann. Rheum. Dis. 68, 1247-1254 (2009).
20. Frank, K. M. et al. Investigation of the cause of death in a gene-therapy trial. N. Engl. J. Med. 361, 161-169 (2009).
21. Gibbons, L. J. & Hyrich, K. L. Biologic therapy for rheumatoid arthritis: clinical efficacy and predictors of response. BioDrugs 23, 111-124 (2009).
22. Tomita, N., Morishita, R., Tomita, T. & Ogihara, T. Potential therapeutic applications of decoy oligonucleotides. Curr. Opin. Mol. Ther. 4, 166-170 (2002) (Pubitemid 34649807)
23. Safety study of TissueGene-C in degenerative joint disease of the knee (TGC-03-01). ClinicalTrials.gov identifier: NCT00599248 [online], http://clinicaltrials.gov/ct2/show/NCT00599248 (2010).
24. Pelletier, J. P et al. In vivo suppression of early experimental osteoarthritis by interleukin-1 receptor antagonist using gene therapy Arthritis Rheum. 40, 1012-1019 (1997).
25. Zhang, X., Mao, Z. & Yu, C Suppression of early experimental osteoarthritis by gene transfer of interleukin-1 receptor antagonist and interleukin-10. J. Orthop. Res. 22, 742-750 (2004). (Pubitemid 38756252)
26. Frisbie, D. D., Ghivizzani, S. C., Robbins, P D., Evans, C H. & McIlwraith, C W. Treatment of experimental equine osteoarthritis by in vivo delivery of the equine interleukin-1 receptor antagonist gene. Gene Ther. 9, 12-20 (2002). (Pubitemid 34145163)
27. Sun, J. et al. Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX. Blood 112, 4532-4541 (2008).
28. Manno, C S. et al. Successful transduction of iver in hemophilia by AAV-Factor IX and imitations imposed by the host immune response. Nat Med. 12, 342-347 (2006). (Pubitemid 43355084)
29. Tyler, W. K., Vidal, A. F., Williams, R. J. & Healey, J. H. Pigmented villonodular synovitis. J. Am. Acad. Orthop. Surg. 14, 376-385 (2006). (Pubitemid 44916652)
30. Byers, S., Rothe, M., Lalic, J., Koldej, R. & Anson, D. S. Lentiviral-mediated correction of MPS VI cells and gene transfer to joint tissues. Mol. Genet. Metab. 97, 102-108 (2009).
31. Evans, C H. Arthritis gene therapy at an inflection point. Future Rheumatol. 3, 207-210 (2008). (Pubitemid 351984097)
32. [No authors listed] Gene therapy deserves a fresh chance. Nature 461, 1173 (2009).