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Research Papers

Preclinical safety evaluation of recombinant adeno-associated virus 2 vector encoding human tumor necrosis factor receptor-immunoglobulin Fc fusion gene

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Pages 732-739 | Received 29 Jun 2015, Accepted 31 Aug 2015, Published online: 05 May 2016

References

  • Woolf AD, Pfleger B. Burden of major musculoskeletal conditions. Bull World Health Organ 2003; 81:646-56; PMID:14710506
  • Zeng QY, Chen R, Darmawan J, Xiao ZY, Chen SB, Wigley R, Le Chen S, Zhang NZ. Rheumatic diseases in China. Arthritis Res Ther 2008; 10:R17; PMID:18237382; http://dx.doi.org/10.1186/ar2368
  • Feldmann M, Maini RN. The role of cytokines in the pathogenesis of rheumatoid arthritis. Rheumatology (Oxford) 1999; 38 Suppl 2:3-7; PMID:10646481
  • Brennan FM, McInnes IB. Evidence that cytokines play a role in rheumatoid arthritis. J Clin Invest 2008; 118:3537-45; PMID:18982160; http://dx.doi.org/10.1172/JCI36389
  • Bathon JM, Martin RW, Fleischmann RM, Tesser JR, Schiff MH, Keystone EC, Genovese MC, Wasko MC, Moreland LW, Weaver AL, et al. A comparison of etanercept and methotrexate in patients with early rheumatoid arthritis. N Engl J Med 2000; 343:1586-93; PMID:11096165; http://dx.doi.org/10.1056/NEJM200011303432201
  • Lipsky PE, van der Heijde DM, St Clair EW, Furst DE, Breedveld FC, Kalden JR, Smolen JS, Weisman M, Emery P, Feldmann M, et al. Infliximab and methotrexate in the treatment of rheumatoid arthritis. Anti-tumor necrosis factor trial in rheumatoid arthritis with concomitant therapy study group. N Engl J Med 2000; 343:1594-602; PMID:11096166; http://dx.doi.org/10.1056/NEJM200011303432202
  • Gomez-Reino JJ, Carmona L, Valverde VR, Mola EM, Montero MD. Treatment of rheumatoid arthritis with tumor necrosis factor inhibitors may predispose to significant increase in tuberculosis risk: a multicenter active-surveillance report. Arthritis Rheum 2003; 48:2122-7; PMID:12905464; http://dx.doi.org/10.1002/art.11137
  • Askling J, Fored CM, Brandt L, Baecklund E, Bertilsson L, Coster L, Geborek P, Jacobsson LT, Lindblad S, Lysholm J, et al. Risk and case characteristics of tuberculosis in rheumatoid arthritis associated with tumor necrosis factor antagonists in Sweden. Arthritis Rheum 2005; 52:1986-92; PMID:15986370; http://dx.doi.org/10.1002/art.21137
  • Woolacott N, Bravo Vergel Y, Hawkins N, Kainth A, Khadjesari Z, Misso K, Light K, Asseburg C, Palmer S, Claxton K, et al. Etanercept and infliximab for the treatment of psoriatic arthritis: a systematic review and economic evaluation. Health Technol Assess 2006; 10:iii-iv, xiii-xvi, 1-239; PMID:17083854
  • Evans CH, Ghivizzani SC, Kang R, Muzzonigro T, Wasko MC, Herndon JH, Robbins PD. Gene therapy for rheumatic diseases. Arthritis Rheum 1999; 42:1-16; PMID:9920008; http://dx.doi.org/10.1002/1529-0131(199901)42:1%3c1::AID-ANR1%3e3.0.CO;2-4
  • Evans CH, Robbins PD, Ghivizzani SC, Wasko MC, Tomaino MM, Kang R, Muzzonigro TA, Vogt M, Elder EM, Whiteside TL, et al. Gene transfer to human joints: progress toward a gene therapy of arthritis. Proc Natl Acad Sci U S A 2005; 102:8698-703; PMID:15939878; http://dx.doi.org/10.1073/pnas.0502854102
  • Bandara G, Mueller GM, Galea-Lauri J, Tindal MH, Georgescu HI, Suchanek MK, Hung GL, Glorioso JC, Robbins PD, Evans CH. Intraarticular expression of biologically active interleukin 1-receptor-antagonist protein by ex vivo gene transfer. Proc Natl Acad Sci U S A 1993; 90:10764-8; PMID:8248169; http://dx.doi.org/10.1073/pnas.90.22.10764
  • Yao Q, Seol DW, Mi Z, Robbins PD. Intra-articular injection of recombinant TRAIL induces synovial apoptosis and reduces inflammation in a rabbit knee model of arthritis. Arthritis Res Ther 2006; 8:R16; PMID:16507116; http://dx.doi.org/10.1186/ar1867
  • Gouze JN, Gouze E, Palmer GD, Liew VS, Pascher A, Betz OB, Thornhill TS, Evans CH, Grodzinsky AJ, Ghivizzani SC. A comparative study of the inhibitory effects of interleukin-1 receptor antagonist following administration as a recombinant protein or by gene transfer. Arthritis Res Ther 2003; 5:R301-9; PMID:12932294; http://dx.doi.org/10.1186/ar795
  • Imagawa T, Watanabe S, Katakura S, Boivin GP, Hirsch R. Gene transfer of a fibronectin peptide inhibits leukocyte recruitment and suppresses inflammation in mouse collagen-induced arthritis. Arthritis Rheum 2002; 46:1102-8; PMID:11953990; http://dx.doi.org/10.1002/art.10188
  • Apparailly F, Millet V, Noel D, Jacquet C, Sany J, Jorgensen C. Tetracycline-inducible interleukin-10 gene transfer mediated by an adeno-associated virus: application to experimental arthritis. Hum Gene Ther 2002; 13:1179-88; PMID:12133271; http://dx.doi.org/10.1089/104303402320138961
  • 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; 6:727-36; PMID:12498769; http://dx.doi.org/10.1006/mthe.2002.0808
  • Zhou X, Gao K, Shen L, Zhao A, Wu X, Wang C, Wang J, Li B. Modulation of immune and inflammatory responses on experimental arthritis following intraarticular gene transfer of tumor necrosis factor receptor-immunoglobulin Fc. Rheumatol Int 2012; 32:2605-14; PMID:21833532; http://dx.doi.org/10.1007/s00296-011-1974-z
  • Verdier F, Descotes J. Preclinical safety evaluation of human gene therapy products. Toxicol Sci 1999; 47:9-15; PMID:10048148; http://dx.doi.org/10.1093/toxsci/47.1.9
  • Huo Y, Li B, Zhang Y, Wang S, Bao M, Gao X, Li D, Wang L, Yu Y, Wang J. Pre-clinical safety evaluation of heat shock protein 65-MUC1 peptide fusion protein. Regul Toxicol Pharmacol 2007; 49:63-74; PMID:17600604; http://dx.doi.org/10.1016/j.yrtph.2007.05.005
  • Peng J, Tan S, Dong X, Wu Z, Yuan H, Chen F, Wu X. Study on transduction of mammal ian cells with recombinant adeno-associated virus 2 in vitro. Chinese J Virol 2004; 20:128-32
  • Wang X, Miao Y, Zhou X, Shen L, Li B. Biodistribution of gag gene transferred by adenovirus vector in mice. Chin J Biologicals 2008; 21:938-40
  • San Sebastian W, Kells AP, Bringas J, Samaranch L, Hadaczek P, Ciesielska A, Macayan M, Pivirotto PJ, Forsayeth J, Osborne S, et al. Safety and tolerability of Mri-guided infusion of AAV2-hAADC into the midbrain of non-human primate. Mol Ther Methods Clin Dev 2014; 3: 14049; PMID:25541617; http://dx.doi.org/10.1038/mtm.2014.49
  • Conrad CK, Allen SS, Afione SA, Reynolds TC, Beck SE, Fee-Maki M, Barrazza-Ortiz X, Adams R, Askin FB, Carter BJ, et al. Safety of single-dose administration of an adeno-associated virus (AAV)-CFTR vector in the primate lung. Gene Ther 1996; 3:658-68; PMID:8854091
  • Guenzel AJ, Hillestad ML, Matern D, Barry MA. Effects of adeno-associated virus serotype and tissue-specific expression on circulating biomarkers of propionic acidemia. Hum Gene Ther 2014; 25:837-43; PMID:25046265; http://dx.doi.org/10.1089/hum.2014.012
  • Davidoff AM, Ng CY, Zhou J, Spence Y, Nathwani AC. Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood 2003; 102:480-8; PMID:12637328; http://dx.doi.org/10.1182/blood-2002-09-2889
  • Gohda T, Niewczas MA, Ficociello LH, Walker WH, Skupien J, Rosetti F, Cullere X, Johnson AC, Crabtree G, Smiles AM, et al. Circulating TNF receptors 1 and 2 predict stage 3 CKD in type 1 diabetes. J Am Soc Nephrol 2012; 23:516-24; PMID:22266664; http://dx.doi.org/10.1681/ASN.2011060628
  • Palanisamy R, Kumaresan V, Harikrishnan R, Arasu MV, Al-Dhabi NA, Arockiaraj J. Functional roles and gene regulation of tumor necrosis factor receptor 1 in freshwater striped murrel. Mol Immunol 2015; 66:240-52; PMID:25841174; http://dx.doi.org/10.1016/j.molimm.2015.03.015
  • Himmler A, Maurer-Fogy I, Kronke M, Scheurich P, Pfizenmaier K, Lantz M, Olsson I, Hauptmann R, Stratowa C, Adolf GR. Molecular cloning and expression of human and rat tumor necrosis factor receptor chain (p60) and its soluble derivative, tumor necrosis factor-binding protein. DNA Cell Biol 1990; 9:705-15; PMID:1702293; http://dx.doi.org/10.1089/dna.1990.9.705
  • Moreland LW, Baumgartner SW, Schiff MH, Tindall EA, Fleischmann RM, Weaver AL, Ettlinger RE, Cohen S, Koopman WJ, Mohler K, et al. Treatment of rheumatoid arthritis with a recombinant human tumor necrosis factor receptor (p75)-Fc fusion protein. N Engl J Med 1997; 337:141-7; PMID:9219699; http://dx.doi.org/10.1056/NEJM199707173370301
  • McPhee SW, Janson CG, Li C, Samulski RJ, Camp AS, Francis J, Shera D, Lioutermann L, Feely M, Freese A, et al. Immune responses to AAV in a phase I study for Canavan disease. J Gene Med 2006; 8:577-88; PMID:16532510; http://dx.doi.org/10.1002/jgm.885
  • Mease PJ, Hobbs K, Chalmers A, El-Gabalawy H, Bookman A, Keystone E, Furst DE, Anklesaria P, Heald AE. 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; 68:1247-54; PMID:18678578; http://dx.doi.org/10.1136/ard.2008.089375
  • Mease PJ, Wei N, Fudman EJ, Kivitz AJ, Schechtman J, Trapp RG, Hobbs KF, Greenwald M, Hou A, Bookbinder SA, 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; 37:692-703; PMID:20032102; http://dx.doi.org/10.3899/jrheum.090817
  • Chirmule N, Xiao W, Truneh A, Schnell MA, Hughes JV, Zoltick P, Wilson JM. Humoral immunity to adeno-associated virus type 2 vectors following administration to murine and nonhuman primate muscle. J Virol 2000; 74:2420-5; PMID:10666273; http://dx.doi.org/10.1128/JVI.74.5.2420-2425.2000
  • Sheets RL, Stein J, Bailer RT, Koup RA, Andrews C, Nason M, He B, Koo E, Trotter H, Duffy C, et al. Biodistribution and toxicological safety of adenovirus type 5 and type 35 vectored vaccines against human immunodeficiency virus-1 (HIV-1), Ebola, or Marburg are similar despite differing adenovirus serotype vector, manufacturer's construct, or gene inserts. J Immunotoxicol 2008; 5:315-35; PMID:18830892; http://dx.doi.org/10.1080/15376510802312464
  • Mori S, Takeuchi T, Enomoto Y, Kondo K, Sato K, Ono F, Iwata N, Sata T, Kanda T. Biodistribution of a low dose of intravenously administered AAV-2, 10, and 11 vectors to cynomolgus monkeys. Jpn J Infect Dis 2006; 59:285-93; PMID:17060693
  • Tatsis N, Fitzgerald JC, Reyes-Sandoval A, Harris-McCoy KC, Hensley SE, Zhou D, Lin SW, Bian A, Xiang ZQ, Iparraguirre A, et al. Adenoviral vectors persist in vivo and maintain activated CD8+ T cells: implications for their use as vaccines. Blood 2007; 110:1916-23; PMID:17510320; http://dx.doi.org/10.1182/blood-2007-02-062117
  • Puntel M, A KMG, Farrokhi C, Vanderveen N, Paran C, Appelhans A, Kroeger KM, Salem A, Lacayo L, Pechnick RN, et al. Safety profile, efficacy, and biodistribution of a bicistronic high-capacity adenovirus vector encoding a combined immunostimulation and cytotoxic gene therapy as a prelude to a phase I clinical trial for glioblastoma. Toxicol Appl Pharmacol 2013; 268:318-30; PMID:23403069; http://dx.doi.org/10.1016/j.taap.2013.02.001
  • Chulay JD, Ye GJ, Thomas DL, Knop DR, Benson JM, Hutt JA, Wang G, Humphries M, Flotte TR. Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method. Hum Gene Ther 2011; 22:155-65; PMID:20812844; http://dx.doi.org/10.1089/hum.2010.118

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