Gene therapy for diabetic retinopathy in animal models and humans

Open Diabetes Journal

Volumn 4, Issue SPEC. ISSUE 1, 2011, Pages 119-122

  Mizukami, Hiroaki ^a   Urabe, Masashi ^a   Kume, Akihiro ^a   Ozawa, Keiya ^a  

^a JICHI MEDICAL UNIVERSITY   (Japan)

Author keywords

AAV vector; Diabetic retinopathy; Gene therapy; SDT rat

Indexed keywords

PARVOVIRUS VECTOR; SMALL INTERFERING RNA; VASCULOTROPIN; VASCULOTROPIN ANTIBODY;

ARTICLE; DIABETIC RETINOPATHY; DISEASE MODEL; DRUG EFFICACY; DRUG RESEARCH; DRUG SAFETY; EXPERIMENTAL RAT; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; HUMAN; NONHUMAN; PRIORITY JOURNAL; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

EID: 80455130998     PISSN: None     EISSN: 18765246     Source Type: Journal    
DOI: 10.2174/1876524601104010119     Document Type: Article

References (30)

1. Eberling JL, Jagust WJ, Christine CW, et al. Results from a phase I safety trial of hAADC gene therapy for Parkinson disease. Neurology 2008; 70: 1980-3.
2. Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 2007; 369: 2097-105.
3. Marks WJ, Jr., Ostrem JL, Verhagen L, et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol 2008; 7: 400-8.
4. Muramatsu S, Fujimoto K, Kato S, et al. A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease. Mol Ther 2010; 18: 1731-5.
5. Bennicelli J, Wright JF, Komaromy A, et al. Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer. Mol Ther 2008; 16: 458-65.
6. Maguire AM, High KA, Auricchio A, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009; 374: 1597-605.
7. Mancuso K, Hauswirth WW, Li Q, et al. Gene therapy for red-green colour blindness in adult primates. Nature 2009; 461: 784-7.
8. Ting JH, Martin DK. Basic and clinical aspects of gene therapy for retinopathy induced by diabetes. Curr Gene Ther 2006; 6: 193-214.
9. Bressler SB. Introduction: Understanding the role of angiogenesis and antiangiogenic agents in age-related macular degeneration. Ophthalmology 2009; 116: S1-7.
10. Kendall RL, Thomas KA. Inhibition of vascular endothelial cell growth factor activity by an endogenously encoded soluble receptor. Proc Natl Acad Sci U S A 1993; 90: 10705-9.
11. Bainbridge JW, Mistry A, De Alwis M, et al. Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt- 1. Gene Ther 2002; 9: 320-6.
12. Honda M, Sakamoto T, Ishibashi T, Inomata H, Ueno H. Experimental subretinal neovascularization is inhibited by adenovirus-mediated soluble VEGF/flt-1 receptor gene transfection: a role of VEGF and possible treatment for SRN in age-related macular degeneration. Gene Ther 2000; 7: 978-85.
13. Lamartina S, Cimino M, Roscilli G, et al. Helper-dependent adenovirus for the gene therapy of proliferative retinopathies: stable gene transfer, regulated gene expression and therapeutic efficacy. J Gene Med 2007; 9: 862-74.
14. Campochiaro PA, Nguyen QD, Shah SM, et al. Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial. Hum Gene Ther 2006; 17: 167-76.
15. Gragoudas ES, Adamis AP, Cunningham ET, Feinsod M, Guyer DR. Pegaptanib for neovascular age-related macular degeneration. N Engl J Med 2004; 351: 2805-16.
16. Murakami Y, Ikeda Y, Yonemitsu Y, et al. Inhibition of choroidal neovascularization via brief subretinal exposure to a newly developed lentiviral vector pseudotyped with Sendai viral envelope proteins. Hum Gene Ther 2010; 21: 199-209.
17. Lee S, Chen TT, Barber CL, et al. Autocrine VEGF signaling is required for vascular homeostasis. Cell 2007; 130: 691-703.
18. Tenenbaum L, Chtarto A, Lehtonen E, Velu T, Brotchi J, Levivier M. Recombinant AAV-mediated gene delivery to the central nervous system. J Gene Med 2004; 6(Suppl 1): S212-22.
19. Lotery AJ, Yang GS, Mullins RF, et al. Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina. Hum Gene Ther 2003; 14: 1663-71.
20. Semple-Rowland SL, Coggin WE, Geesey M, et al. Expression characteristics of dual-promoter lentiviral vectors targeting retinal photoreceptors and Muller cells. Mol Vis 2010; 16: 916-34.
21. Dalkara D, Kolstad KD, Caporale N, et al. Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous. Mol Ther 2009; 17: 2096-102.
22. Connor KM, Krah NM, Dennison RJ, et al. Quantification of oxygen-induced retinopathy in the mouse: a model of vessel loss, vessel regrowth and pathological angiogenesis. Nat Protoc 2009; 4: 1565-73.
23. Kador PF, Takahashi Y, Wyman M, Ferris F. Diabeteslike proliferative retinal changes in galactose-fed dogs. Arch Ophthalmol 1995; 113: 352-4.
24. Engerman RL, Kern TS. Retinopathy in galactosemic dogs continues to progress after cessation of galactosemia. Arch Ophthalmol 1995; 113: 355-8.
25. Kim SY, Johnson MA, McLeod DS, et al. Retinopathy in monkeys with spontaneous type 2 diabetes. Invest Ophthalmol Vis Sci 2004; 45: 4543-53.
26. Lebherz C, Maguire AM, Auricchio A, et al. Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor. Diabetes 2005; 54: 1141-9.
27. Shinohara M, Masuyama T, Shoda T, et al. A new spontaneously diabetic non-obese Torii rat strain with severe ocular complications. Int J Exp Diabetes Res 2000; 1: 89-100.
28. Kakehashi A, Saito Y, Mori K, et al. Characteristics of diabetic retinopathy in SDT rats. Diabetes Metab Res Rev 2006; 22: 455-61.
29. Ideno J, Mizukami H, Kakehashi A, et al. Prevention of diabetic retinopathy by intraocular soluble flt-1 gene transfer in a spontaneously diabetic rat model. Int J Mol Med 2007; 19: 75-9.
30. Hasumi Y, Mizukami H, Urabe M, et al. Soluble FLT-1 expression suppresses carcinomatous ascites in nude mice bearing ovarian cancer. Cancer Res 2002; 62: 2019-23.