Arthritis gene therapy trials reach phase II

Journal of Rheumatology

Volumn 37, Issue 4, 2010, Pages 683-685

  Evans, Christopher ^a  

^a BETH ISRAEL DEACONESS MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; DISEASE MODIFYING ANTIRHEUMATIC DRUG; ETANERCEPT; PLACEBO; TUMOR NECROSIS FACTOR ALPHA INHIBITOR; TUMOR NECROSIS FACTOR ALPHA INHIBITOR FUSION PROTEIN; UNCLASSIFIED DRUG;

CLINICAL FEATURE; DRUG EFFICACY; DRUG FATALITY; DRUG SAFETY; EDITORIAL; GENE THERAPY; GENE TRANSFER; HISTOPLASMOSIS; HUMAN; IMMUNE RESPONSE; LUNG EMBOLISM; OUTCOME ASSESSMENT; PRIORITY JOURNAL; RETROPERITONEAL HEMATOMA; RHEUMATOID ARTHRITIS; TREATMENT RESPONSE; VIRUS GENOME;

ARTHRITIS; CLINICAL TRIALS, PHASE II AS TOPIC; GENE THERAPY; HUMANS;

EID: 77950663420     PISSN: 0315162X     EISSN: None     Source Type: Journal    
DOI: 10.3899/jrheum.100055     Document Type: Editorial

References (22)

1. Bandara G, Robbins PD, Georgescu HI, Mueller GM, Glorioso JC, Evans CH. Gene transfer to synoviocytes: prospects for gene treatment of arthritis. DNA Cell Biol 1992;11:227-231
2. Evans CH, Ghivizzani SC, Robbins PD. Gene therapy for arthritis: what next? Arthritis Rheum 2006;54:1714-1729
3. Evans CH, Ghivizzani SC, Robbins PD. Gene therapy of the rheumatic diseases: 1998 to 2008. Arthritis Res Ther 2009;11:209.
4. Evans CH. Gene therapy at an inflection point. Future Rheumatol 2008;3:207-210
5. Evans CH, Robbins PD, Ghivizzani SC, Wasko MC, Tomaino MM, Kang R, et al. Gene transfer to human joints: progress toward a gene therapy of arthritis. Proc Natl Acad Sci USA 2005;102:8698-8703 (Pubitemid 40862798)
6. Mease PJ, Hobbs K, Chalmers A, El-Gabalawy H, Bookman A, Keystone E, et al. Local delivery of a recombinant adenoassociated vector containing a tumour necrosis factor alpha antagonist gene in inflammatory arthritis: a phase 1 dose-escalation safety and tolerability study. Ann Rheum Dis 2009;68:1247-1254
7. Wehling P, Reinecke J, Baltzer AA, Granrath M, Schulitz KP, Schultz C, et al. Clinical responses to gene therapy in joints of two subjects with rheumatoid arthritis. Hum Gene Ther 2009; 20:97-101.
8. Mease PJ, Wei N, Fudman EJ, Kivitz AJ, Schechtman J, Trapp RG, 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 2010;37:692-704.
9. Ghivizzani SC, Lechman ER, Kang R, Tio C, Kolls J, Evans CH, 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 (Pubitemid 28207961)
10. Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 2006;14:316-327 (Pubitemid 44184972)
11. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008;358:2231-2239 (Pubitemid 351724452)
12. Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008;358:2240-2248 (Pubitemid 351724453)
13. Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006;12:342-347 (Pubitemid 43355084)
14. Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, et al. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 2007;13:419-422 (Pubitemid 46559826)
15. Chan JM, Villarreal G, Jin WW, Stepan T, Burstein H, Wahl SM. Intraarticular gene transfer of TNFR:Fc suppresses experimental arthritis with reduced systemic distribution of the gene product. Mol Ther 2002;6:727-736 (Pubitemid 36054306)
16. Frank KM, Hogarth DK, Miller JL, Mandal S, Mease PJ, Samulski RJ, et al. Investigation of the cause of death in a gene-therapy trial. N Engl J Med 2009;361:161-169
17. Evans CH, Ghivizzani SC, Robbins PD. Arthritis gene therapy's first death. Arthritis Res Ther 2008;10:110.
18. Kay JD, Gouze E, Oligino TJ, Gouze JN, Watson RS, Levings PP, et al. Intra-articular gene delivery and expression of interleukin-1Ra mediated by self-complementary adeno-associated virus. J Gene Med 2009;11:605-614
19. Apparailly F, Khoury M, Vervoordeldonk MJ, Adriaansen J, Gicquel E, Perez N, et al. Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints. Hum Gene Ther 2005;16:426-434 (Pubitemid 40647140)
20. Boissier MC, Lemeiter D, Clavel C, Valvason C, Laroche L, Begue T, et al. Synoviocyte infection with adeno-associated virus (AAV) is neutralized by human synovial fluid from arthritis patients and depends on AAV serotype. Hum Gene Ther 2007;18:525-535 (Pubitemid 47036046)
21. Kolata G. After setbacks, small successes for gene therapy. New York Times; Nov 5 2009.
22. Gene therapy deserves a fresh chance [editorial]. Nature 2009;461:1173.