Adeno-associated virus type 8 vector-mediated expression of siRNA targeting vascular endothelial growth factor efficiently inhibits neovascularization in a murine choroidal neovascularization model

Molecular Vision

Volumn 20, Issue , 2014, Pages 488-496

  Igarashi, Tsutomu ^a   Miyake, Noriko ^a   Fujimoto, Chiaki ^a   Yaguchi, Chiemi ^a   Iijima, Osamu ^a   Shimada, Takashi ^a   Takahashi, Hiroshi ^a   Miyake, Koichi ^a  

^a NIPPON MEDICAL SCHOOL   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

GREEN FLUORESCENT PROTEIN; PARVOVIRUS VECTOR; SMALL INTERFERING RNA; VASCULOTROPIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIANGIOGENIC ACTIVITY; ANTIANGIOGENIC GENE THERAPY; ANTIANGIOGENIC THERAPY; ARTICLE; CONTROLLED STUDY; DISEASE MODEL; FEASIBILITY STUDY; GENE EXPRESSION; GENE REPRESSION; GENE TARGETING; IN VIVO GENE TRANSFER; MALE; MOUSE; NONHUMAN; PIGMENT CELL; PRIORITY JOURNAL; SUBRETINAL NEOVASCULARIZATION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

3T3 CELLS; ANIMALS; BASE SEQUENCE; CHOROID; CHOROIDAL NEOVASCULARIZATION; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HUMANS; MICE; MICE, INBRED C57BL; MOLECULAR SEQUENCE DATA; RETINAL PIGMENT EPITHELIUM; RNA, SMALL INTERFERING; TRANSDUCTION, GENETIC; TRANSFECTION; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 84898410131     PISSN: None     EISSN: 10900535     Source Type: Journal    
DOI: None     Document Type: Article

References (42)

1. Hotchkiss ML, Fine SL. Pathologic myopia and choroidal neovascularization. Am J Ophthalmol 1981; 91:177-83. [PMID: 6162388].
2. Sickenberg M, Schmidt-Erfurth U, Miller JW, Pournaras CJ, Zografos L, Piguet B, Donati G, Laqua H, Barbazetto I, Gragoudas ES, Lane AM, Birngruber R, van den Bergh H, Strong HA, Manjuris U, Gray T, Fsadni M, Bressler NM. A preliminary study of photodynamic therapy using verteporfin for choroidal neovascularization in pathologic myopia, ocular histoplasmosis syndrome, angioid streaks, and idiopathic causes. Arch Ophthalmol 2000; 118:327-36. [PMID: 10721954].
3. Young RW. Pathophysiology of age-related macular degeneration. Surv Ophthalmol 1987; 31:291-306. [PMID: 3299827].
4. Starr CE, Guyer DR, Yannuzzi LA. Age-related macular degeneration. Can we stem this worldwide public health crisis? Postgrad Med 1998; 103:153-6. [PMID: 9590992].
5. Congdon N, O'Colmain B, Klaver CC, Klein R, Munoz B, Friedman DS, Kempen J, Taylor HR, Mitchell P. Causes and prevalence of visual impairment among adults in the United States. Arch Ophthalmol 2004; 122:477-85. [PMID: 15078664].
6. Coleman HR, Chan CC, Ferris FL 3rd, Chew EY. Age-related macular degeneration. Lancet 2008; 372:1835-45. [PMID: 19027484].
7. Senger DR, Galli SJ, Dvorak AM, Perruzzi CA, Harvey VS, Dvorak HF. Tumor cells secrete a vascular permeability factor that promotes accumulation of ascites fluid. Science 1983; 219:983-5. [PMID: 6823562].
8. Keck PJ, Hauser SD, Krivi G, Sanzo K, Warren T, Feder J, Connolly DT. Vascular permeability factor, an endothelial cell mitogen related to PDGF. Science 1989; 246:1309-12. [PMID: 2479987].
9. Ferrara N, Houck KA, Jakeman LB, Winer J, Leung DW. The vascular endothelial growth factor family of polypeptides. J Cell Biochem 1991; 47:211-8. [PMID: 1791185].
10. Alon T, Hemo I, Itin A, Pe'er J, Stone J, Keshet E. Vascular endothelial growth factor acts as a survival factor for newly formed retinal vessels and has implications for retinopathy of prematurity. Nat Med 1995; 1:1024-8. [PMID: 7489357].
11. Shweiki D, Itin A, Soffer D, Keshet E. Vascular endothelial growth factor induced by hypoxia may mediate hypoxia-initiated angiogenesis. Nature 1992; 359:843-5. [PMID: 1279431].
12. Cheng T, Cao W, Wen R, Steinberg RH, LaVail MM. Prostaglandin E2 induces vascular endothelial growth factor and basic fibroblast growth factor mRNA expression in cultured rat Muller cells. Invest Ophthalmol Vis Sci 1998; 39:581-91. [PMID: 9501870].
13. Kvanta A, Algvere PV, Berglin L, Seregard S. Subfoveal fibrovascular membranes in age-related macular degeneration express vascular endothelial growth factor. Invest Ophthalmol Vis Sci 1996; 37:1929-34. [PMID: 8759365].
14. Hera R, Keramidas M, Peoc'h M, Mouillon M, Romanet JP, Feige JJ. Expression of VEGF and angiopoietins in subfoveal membranes from patients with age-related macular degeneration. Am J Ophthalmol 2005; 139:589-96. [PMID: 15808152].
15. Yi X, Ogata N, Komada M, Yamamoto C, Takahashi K, Omori K, Uyama M. Vascular endothelial growth factor expression in choroidal neovascularization in rats. Graefes Arch Clin Exp Ophthalmol 1997; 235:313-9. [PMID: 9176680].
16. de Vries C, Escobedo JA, Ueno H, Houck K, Ferrara N, Williams LT. The fms-like tyrosine kinase, a receptor for vascular endothelial growth factor. Science 1992; 255:989-91. [PMID: 1312256].
17. Mustonen T, Alitalo K. Endothelial receptor tyrosine kinases involved in angiogenesis. J Cell Biol 1995; 129:895-8. [PMID: 7538139].
18. Igarashi T, Miyake K, Masuda I, Takahashi H, Shimada T. Adeno-associated vector (type 8)-mediated expression of soluble Flt-1 efficiently inhibits neovascularization in a murine choroidal neovascularization model. Hum Gene Ther 2010; 21:631-7. [PMID: 20053138].
19. Gragoudas ES, Adamis AP, Cunningham ET Jr, Feinsod M, Guyer DR. Pegaptanib for neovascular age-related macular degeneration. N Engl J Med 2004; 351:2805-16. [PMID: 15625332].
20. Ng EW, Shima DT, Calias P, Cunningham ET Jr, Guyer DR, Adamis AP. Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease. Nat Rev Drug Discov 2006; 5:123-32. [PMID: 16518379].
21. Rosenfeld PJ, Brown DM, Heier JS, Boyer DS, Kaiser PK, Chung CY, Kim RY. Ranibizumab for neovascular age-related macular degeneration. N Engl J Med 2006; 355:1419-31. [PMID: 17021318].
22. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411:494-8. [PMID: 11373684].
23. Reich SJ, Fosnot J, Kuroki A, Tang W, Yang X, Maguire AM, Bennett J, Tolentino MJ. Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. Mol Vis 2003; 9:210-6. [PMID: 12789138].
24. Shen J, Samul R, Silva RL, Akiyama H, Liu H, Saishin Y, Hackett SF, Zinnen S, Kossen K, Fosnaugh K, Vargeese C, Gomez A, Bouhana K, Aitchison R, Pavco P, Campochiaro PA. Suppression of ocular neovascularization with siRNA targeting VEGF receptor 1. Gene Ther 2006; 13:225-34. [PMID: 16195704].
25. Miyake K, Miyake N, Yamazaki Y, Shimada T, Hirai Y. Serotype-independent method of recombinant adeno-associated virus (AAV) vector production and purification. J Nippon Med Sch 2012; 79:394-402. [PMID: 23291836].
26. Askou AL, Pournaras JA, Pihlmann M, Svalgaard JD, Arsenijevic Y, Kostic C, Bek T, Dagnaes-Hansen F, Mikkelsen JG, Jensen TG, Corydon TJ. Reduction of choroidal neovascularization in mice by adeno-associated virus-delivered anti-vascular endothelial growth factor short hairpin RNA. J Gene Med 2012; 14:632-41. [PMID: 23080553].
27. Naito Y, Yamada T, Ui-Tei K, Morishita S, Saigo K. siDirect: highly effective, target-specific siRNA design software for mammalian RNA interference. Nucleic Acids Res 2004; 32:W124-9-[PMID: 15215364].
28. Hao DL, Liu CM, Dong WJ, Gong H, Wu XS, Liu DP, Liang CC. Knockdown of human p53 gene expression in 293-T cells by retroviral vector-mediated short hairpin RNA. Acta Biochim Biophys Sin (Shanghai) 2005; 37:779-83. [PMID: 16270158].
29. Ro S, Hwang SJ, Ordög T, Sanders KM. Adenovirus-based short hairpin RNA vectors containing an EGFP marker and mouse U6, human H1, or human U6 promoter. Biotechniques 2005; 38:625-7. [PMID: 15884680].
30. Takahashi H, Kato K, Miyake K, Hirai Y, Yoshino S, Shimada T. Adeno-associated virus vector-mediated anti-angiogenic gene therapy for collagen-induced arthritis in mice. Clin Exp Rheumatol 2005; 23:455-61. [PMID: 16095112].
31. Igarashi T, Miyake K, Asakawa N, Miyake N, Shimada T, Takahashi H. Direct Comparison of Administration Routes for AAV8-mediated Ocular Gene Therapy. Curr Eye Res 2013; 38:569-77. [PMID: 23489150].
32. Singerman L. Combination therapy using the small interfering RNA bevasiranib. Retina 2009; 29:S49-50. [PMID: 19553802].
33. Kaiser PK, Symons RC, Shah SM, Quinlan EJ, Tabandeh H, Reisen G, Lockridge JA, Short B, Guerciolini R, Nguyen QD. RNAi-based treatment for neovascular age-related macular degeneration by Sirna-027. Am J Ophthalmol 2010; 150:33-9. [PMID: 20609706].
34. Kleinman ME, Yamada K, Takeda A, Chandrasekaran V, Nozaki M, Baffi JZ, Albuquerque RJ, Yamasaki S, Itaya M, Pan Y, Appukuttan B, Gibbs D, Yang Z, Kariko K, Ambati BK, Wilgus TA, DiPietro LA, Sakurai E, Zhang K, Smith JR, Taylor EW, Ambati J. Sequence-and target-independent angiogenesis suppression by siRNA via TLR3. Nature 2008; 452:591-7. [PMID: 18368052].
35. Ashikari M, Tokoro M, Itaya M, Nozaki M, Ogura Y. Suppression of laser-induced choroidal neovascularization by nontargeted siRNA. Invest Ophthalmol Vis Sci 2010; 51:3820-4. [PMID: 20130283].
36. Gu L, Chen H, Tuo J, Gao X, Chen L. Inhibition of experimental choroidal neovascularization in mice by anti-VEGFA/VEGFR2 or non-specific siRNA. Exp Eye Res 2010; 91:433-9. [PMID: 20599960].
37. Kleinman ME, Kaneko H, Cho WG, Dridi S, Fowler BJ, Blandford AD, Albuquerque RJ, Hirano Y, Terasaki H, Kondo M, Fujita T, Ambati BK, Tarallo V, Gelfand BD, Bogdanovich S, Baffi JZ, Ambati J. Short-interfering RNAs induce retinal degeneration via TLR3 and IRF3. Mol Ther 2012; 20:101-8. [PMID: 21988875].
38. Dinculescu A, Glushakova L, Min SH, Hauswirth WW. Adeno-associated virus-vectored gene therapy for retinal disease. Hum Gene Ther 2005; 16:649-63. [PMID: 15960597].
39. Mussolino C, della Corte M, Rossi S, Viola F, Di Vicino U, Marrocco E, Neglia S, Doria M, Testa F, Giovannoni R, Crasta M, Giunti M, Villani E, Lavitrano M, Bacci ML, Ratiglia R, Simonelli F, Auricchio A, Surace EM. AAV-mediated photoreceptor transduction of the pig cone-enriched retina. Gene Ther 2011; 18:637-45. [PMID: 21412286].
40. Justilien V, Pang JJ, Renganathan K, Zhan X, Crabb JW, Kim SR, Sparrow JR, Hauswirth WW, Lewin AS. SOD2 knockdown mouse model of early AMD. Invest Ophthalmol Vis Sci 2007; 48:4407-20. [PMID: 17898259].
41. Stieger K, Colle MA, Dubreil L, Mendes-Madeira A, Weber M, Le Meur G, Deschamps JY, Provost N, Nivard D, Cherel Y, Moullier P, Rolling F. Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain. Mol Ther 2008; 16:916-23. [PMID: 18388922].
42. Igarashi T, Miyake K, Kato K, Watanabe A, Ishizaki M, Ohara K, Shimada T. Lentivirus-mediated expression of angiostatin efficiently inhibits neovascularization in a murine proliferative retinopathy model. Gene Ther 2003; 10:219-26. [PMID: 12571629].