The importance of hypoxia-regulated, RPE-targeted gene therapy for choroidal neovascularization

Advances in Experimental Medicine and Biology

Volumn 723, Issue , 2012, Pages 269-277

  Smith, George W ^a   Dorey, C Kathleen ^b   Prentice, Howard ^c,d   Blanks, Janet ^c,d  

^a UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^b VIRGINIA TECH CARILION SCHOOL OF MEDICINE   (United States)

^c FLORIDA ATLANTIC UNIVERSITY   (United States)

^d FLORIDA ATLANTIC UNIVERSITY   (United States)

Author keywords

Age Related Macular Degeneration; Choroidal Neovascularization; Endostatin; Gene Therapy; HIF 1; Hypoxia; Retinal Pigment Epithelium

Indexed keywords

ANGIOSTATIN; ENDOSTATIN; HYPOXIA INDUCIBLE FACTOR 1; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; PIGMENT EPITHELIUM DERIVED FACTOR; RAPAMYCIN; TETRACYCLINE; VASCULOTROPIN;

APOPTOSIS; CONFERENCE PAPER; DRUG EFFICACY; GENE EXPRESSION; GENE THERAPY; HUMAN; HYPOXIA; INFLAMMATION; NONHUMAN; OXIDATIVE STRESS; PIGMENT EPITHELIUM; PRIORITY JOURNAL; PROMOTER REGION; RETINA DETACHMENT; RETINA MACULA AGE RELATED DEGENERATION; SUBRETINAL NEOVASCULARIZATION; VIRAL GENE DELIVERY SYSTEM;

CHOROIDAL NEOVASCULARIZATION; GENE THERAPY; HUMANS; HYPOXIA-INDUCIBLE FACTOR 1, ALPHA SUBUNIT; MACULAR DEGENERATION; RETINAL PIGMENT EPITHELIUM; WET MACULAR DEGENERATION;

EID: 84862936377     PISSN: 00652598     EISSN: None     Source Type: Book Series    
DOI: 10.1007/978-1-4614-0631-0_35     Document Type: Conference Paper

References (55)

1. Agha-Mohammadi S, O'Malley M, Etemad A et al (2004) Second-generation tetracycline-regulatable promoter: repositioned tet operator elements optimize transactivator synergy while shorter minimal promoter offers tight basal leakiness. J Gene Med 6: 817-828 (Pubitemid 40228098)
2. Ali RR, Sarra GM, Stephens C et al (2000) Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 25: 306-310 (Pubitemid 30437318)
3. Ambati J, Ambati BK, Yoo SH et al (2003) Age-related macular degeneration: etiology, pathogenesis, and therapeutic strategies. Surv Ophthalmol 48: 257-293 (Pubitemid 36553096)
4. Anderson DH, Mullins RF, Hageman GS et al (2002) A role for local infl ammation in the formation of drusen in the aging eye. Am J Ophthalmol 134: 411-431
5. Bainbridge JW, Mistry A, Binley K et al (2003) Hypoxia-regulated transgene expression in experimental retinal and choroidal neovascularization. Gene Ther 10: 1049-1054 (Pubitemid 36722496)
6. Balaggan KS, Binley K, Esapa M et al (2006) EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV. Gene Ther 13: 1153-1165 (Pubitemid 44084074)
7. Bennett J, Zeng Y, Bajwa R et al (1998) Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse. Gene Ther 5: 1156-1164 (Pubitemid 28448422)
8. Binley K, Iqball S, Kingsman A et al (1999) An adenoviral vector regulated by hypoxia for the treatment of ischaemic disease and cancer. Gene Ther 6: 1721-1727 (Pubitemid 29485947)
9. Boulanger A, Liu S, Henningsgaard AA et al (2000) The upstream region of the Rpe65 gene confers retinal pigment epithelium-specifi c expression in vivo and in vitro and contains critical octamer and E-box binding sites. J Biol Chem 275: 31274-31282
10. Caniggia I, Mostachfi H, Winter J et al (2000) Hypoxia-inducible factor-1 mediates the biological effects of oxygen on human trophoblast differentiation through TGFbeta(3). J Clin Invest 105: 577-587 (Pubitemid 30146504)
11. D'Amato RJ, Adamis AP (1995) Angiogenesis inhibition in age-related macular degeneration. Ophthalmology 102: 1261-1262
12. Do DV (2009) Antiangiogenic approaches to age-related macular degeneration in the future. Ophthalmology 116: S24-26
13. Dong Z, Zhou L, Del Villar K et al (2005) JIP1 regulates neuronal apoptosis in response to stress. Brain Res Mol Brain Res 134: 282-293
14. Dorey CK, Wu G, Ebenstein D et al (1989) Cell loss in the aging retina. Relationship to lipofuscin accumulation and macular degeneration. Invest Ophthalmol Vis Sci 30: 1691-1699 (Pubitemid 19214989)
15. Dougherty CJ, Smith GW, Dorey CK et al (2008) Robust hypoxia-selective regulation of a retinal pigment epithelium-specifi c adeno-associated virus vector. Mol Vis 14: 471-480 (Pubitemid 351377319)
16. Eaton D, Gilham DE, O'Neill A et al (2002) Retroviral transduction of human peripheral blood lymphocytes with Bcl-X(L) promotes in vitro lymphocyte survival in pro-apoptotic conditions. Gene Ther 9: 527-535 (Pubitemid 34449773)
17. Fine SL, Berger JW, Maguire MG et al (2000) Age-related macular degeneration. N Engl J Med 342: 483-492 (Pubitemid 30100237)
18. Fraefel C, Mendes-Madeira A, Mabon O et al (2005) In vivo gene transfer to the rat retina using herpes simplex virus type 1 (HSV-1)-based amplicon vectors. Gene Ther 12: 1283-1288 (Pubitemid 41195119)
19. Friedman DS, O'Colmain BJ, Munoz B et al (2004) Prevalence of age-related macular degeneration in the United States. Arch Ophthalmol 122: 564-572 (Pubitemid 38456293)
20. Green ES, Rendahl KG, Zhou S et al (2001) Two animal models of retinal degeneration are rescued by recombinant adeno-associated virus-mediated production of FGF-5 and FGF-18. Mol Ther 3: 507-515 (Pubitemid 32509844)
21. Green WR (1999) Histopathology of age-related macular degeneration. Mol Vis 5: 27
22. Hauswirth WW, LaVail MM, Flannery JG et al (2000) Ribozyme gene therapy for autosomal dominant retinal disease. Clin Chem Lab Med 38: 147-153 (Pubitemid 30245783)
23. Hernandez OM, Discher DJ, Bishopric NH et al (2000) Rapid activation of neutral sphingomyelinase by hypoxia-reoxygenation of cardiac myocytes. Circ Res 86: 198-204 (Pubitemid 30086181)
24. Holz FG, Schutt F, Kopitz J et al (1999) Inhibition of lysosomal degradative functions in RPE cells by a retinoid component of lipofuscin. Invest Ophthalmol Vis Sci 40: 737-743 (Pubitemid 29109453)
25. Johnson LV, Ozaki S, Staples MK et al (2000) A potential role for immune complex pathogenesis in drusen formation. Exp Eye Res 70: 441-449 (Pubitemid 30662042)
26. Jung YJ, Isaacs JS, Lee S et al (2003) IL-1beta-mediated up-regulation of HIF-1alpha via an NFkappaB/COX-2 pathway identifi es HIF-1 as a critical link between infl ammation and oncogenesis. FASEB J 17: 2115-2117
27. Kachi S, Esumi N, Zack DJ et al (2006) Sustained expression after nonviral ocular gene transfer using mammalian promoters. Gene Ther 13: 798-804
28. Kachi S, Binley K, Yokoi K et al (2009) Equine infectious anemia viral vector-mediated codelivery of endostatin and angiostatin driven by retinal pigmented epithelium-specifi c VMD2 promoter inhibits choroidal neovascularization. Hum Gene Ther 20: 31-39
29. Klein R, Peto T, Bird A et al (2004) The epidemiology of age-related macular degeneration. Am J Ophthalmol 137: 486-495 (Pubitemid 38328641)
30. Le Meur G, Stieger K, Smith AJ et al (2007) Restoration of vision in RPE65-defi cient Briard dogs using an AAV serotype 4 vector that specifi cally targets the retinal pigmented epithelium. Gene Ther 14: 292-303 (Pubitemid 46231923)
31. Mandel RJ, Spratt SK, Snyder RO et al (1997) Midbrain injection of recombinant adeno-associated virus encoding rat glial cell line-derived neurotrophic factor protects nigral neurons in a progressive 6-hydroxydopamine-induced degeneration model of Parkinson's disease in rats. Proc Natl Acad Sci U S A 94: 14083-14088
32. Mousa SA, Lorelli W, Campochiaro PA (1999) Role of hypoxia and extracellular matrix-integrin binding in the modulation of angiogenic growth factors secretion by retinal pigmented epithelial cells. J Cell Biochem 74: 135-143 (Pubitemid 29270036)
33. Murakami H, Yayama K, Chao L et al (1998) Human kallikrein gene delivery protects against gentamycin-induced nephrotoxicity in rats. Kidney Int 53: 1305-1313 (Pubitemid 28316892)
34. Ozaki H, Yu AY, Della N et al (1999) Hypoxia inducible factor-1alpha is increased in ischemic retina: temporal and spatial correlation with VEGF expression. Invest Ophthalmol Vis Sci 40: 182-189 (Pubitemid 29029587)
35. Prentice H, Bishopric NH, Hicks MN et al (1997) Regulated expression of a foreign gene targeted to the ischaemic myocardium. Cardiovasc Res 35: 567-574 (Pubitemid 27469420)
36. Robison WG, Jr., Kuwabara T, Bieri JG (1980) Defi ciencies of vitamins E and A in the rat. Retinal damage and lipofuscin accumulation. Invest Ophthalmol Vis Sci 19: 1030-1037
37. Ruan H, Su H, Hu L et al (2001) A hypoxia-regulated adeno-associated virus vector for cancerspecifi c gene therapy. Neoplasia 3: 255-263 (Pubitemid 32747574)
38. Sandau KB, Zhou J, Kietzmann T et al (2001) Regulation of the hypoxia-inducible factor 1alpha by the infl ammatory mediators nitric oxide and tumor necrosis factor-alpha in contrast to desferroxamine and phenylarsine oxide. J Biol Chem 276: 39805-39811
39. Schoenherr CJ, Anderson DJ (1995) The neuron-restrictive silencer factor (NRSF): a coordinate repressor of multiple neuron-specifi c genes. Science 267: 1360-1363
40. Schuettauf F, Vorwerk C, Naskar R et al (2004) Adeno-associated viruses containing bFGF or BDNF are neuroprotective against excitotoxicity. Curr Eye Res 29: 379-386 (Pubitemid 40138377)
41. Semenza GL, Nejfelt MK, Chi SM et al (1991) Hypoxia-inducible nuclear factors bind to an enhancer element located 3 ¢ to the human erythropoietin gene. Proc Natl Acad Sci U S A 88: 5680-5684 (Pubitemid 21914809)
42. Sheridan CM, Pate S, Hiscott P et al (2009) Expression of hypoxia-inducible factor-1alpha and -2alpha in human choroidal neovascular membranes. Graefes Arch Clin Exp Ophthalmol 247: 1361-1367
43. Stiehl DP, Jelkmann W, Wenger RH et al (2002) Normoxic induction of the hypoxia-inducible factor 1alpha by insulin and interleukin-1beta involves the phosphatidylinositol 3-kinase pathway. FEBS Lett 512: 157-162 (Pubitemid 34164471)
44. Takahashi K, Saishin Y, Silva RL et al (2003) Intraocular expression of endostatin reduces VEGF-induced retinal vascular permeability, neovascularization, and retinal detachment. FASEB J 17: 896-898
45. Tang YL, Tang Y, Zhang YC et al (2005) A hypoxia-inducible vigilant vector system for activating therapeutic genes in ischemia. Gene Ther 12: 1163-1170 (Pubitemid 41136310)
46. Vinores SA, Xiao WH, Aslam S et al (2006) Implication of the hypoxia response element of the Vegf promoter in mouse models of retinal and choroidal neovascularization, but not retinal vascular development. J Cell Physiol 206: 749-758 (Pubitemid 43191050)
47. Wassell J, Davies S, Bardsley W et al (1999) The photoreactivity of the retinal age pigment lipofuscin. J Biol Chem 274: 23828-23832
48. Webster KA (2003) Therapeutic angiogenesis: a complex problem requiring a sophisticated approach. Cardiovasc Toxicol 3: 283-298
49. Yang XM, Wang YS, Zhang J et al (2009) Role of PI3K/Akt and MEK/ERK in mediating hypoxiainduced expression of HIF-1alpha and VEGF in laser-induced rat choroidal neovascularization. Invest Ophthalmol Vis Sci 50: 1873-1879
50. Yin H, Chao L, Chao J (2004) Adrenomedullin protects against myocardial apoptosis after ischemia/ reperfusion through activation of Akt-GSK signaling. Hypertension 43: 109-116 (Pubitemid 38031918)
51. Young RW (1988) Solar radiation and age-related macular degeneration. Surv Ophthalmol 32: 252-269 (Pubitemid 18066235)
52. Zarbin MA (2004) Current concepts in the pathogenesis of age-related macular degeneration. Arch Ophthalmol 122: 598-614 (Pubitemid 38456297)
53. Zhang C, Wang YS, Wu H et al (2010) Inhibitory effi cacy of hypoxia-inducible factor 1alpha short hairpin RNA plasmid DNA-loaded poly (D, L-lactide-co-glycolide) nanoparticles on choroidal neovascularization in a laser-induced rat model. Gene Ther 17: 338-351
54. Zhang P, Wang Y, Hui Y et al (2007) Inhibition of VEGF expression by targeting HIF-1 alpha with small interference RNA in human RPE cells. Ophthalmologica 221: 411-417 (Pubitemid 47608868)
55. Zhuo Y, Chen PF, Zhang AZ et al (2009) Cardioprotective effect of hydrogen sulfi de in ischemic reperfusion experimental rats and its infl uence on expression of survivin gene. Biol Pharm Bull 32: 1406-1410