-
2
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
Bainbridge JWB, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008; 358 : 2231-9.
-
(2008)
N Engl J Med
, vol.358
, pp. 2231-2239
-
-
Bainbridge, J.W.B.1
Smith, A.J.2
Barker, S.S.3
-
3
-
-
54449085219
-
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
-
Cideciyan AV, Aleman TS, Boye SL, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci USA 2008; 105 : 15112-7.
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 15112-15117
-
-
Cideciyan, A.V.1
Aleman, T.S.2
Boye, S.L.3
-
4
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008; 358 : 2240-8.
-
(2008)
N Engl J Med
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
-
5
-
-
84897051037
-
Retinal gene therapy in patients with choroideremia: Initial findings from a phase 1/2 clinical trial
-
Maclaren RE, Groppe M, Barnard AR, et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet 2014; 383 : 1129-37.
-
(2014)
Lancet
, vol.383
, pp. 1129-1137
-
-
Maclaren, R.E.1
Groppe, M.2
Barnard, A.R.3
-
6
-
-
50049136340
-
Success in sight: The eyes have it! Ocular gene therapy trials for LCA look promising
-
Bainbridge JW, Ali RR. Success in sight: the eyes have it! Ocular gene therapy trials for LCA look promising. Gene Ther 2008; 15 : 1191-2.
-
(2008)
Gene Ther
, vol.15
, pp. 1191-1192
-
-
Bainbridge, J.W.1
Ali, R.R.2
-
7
-
-
84879606470
-
A review of therapeutic prospects of nonviral gene therapy in the retinal pigment epithelium
-
Koirala A, Conley SM, Naash MI. A review of therapeutic prospects of nonviral gene therapy in the retinal pigment epithelium. Biomaterials 2013; 34 : 7158-67.
-
(2013)
Biomaterials
, vol.34
, pp. 7158-7167
-
-
Koirala, A.1
Conley, S.M.2
Naash, M.I.3
-
8
-
-
84856950131
-
Novel adeno-associated viral vectors for retinal gene therapy
-
Vandenberghe LH, Auricchio A. Novel adeno-associated viral vectors for retinal gene therapy. Gene Ther 2012; 19 : 162-8.
-
(2012)
Gene Ther
, vol.19
, pp. 162-168
-
-
Vandenberghe, L.H.1
Auricchio, A.2
-
9
-
-
79955954680
-
AAV and compacted DNA nanoparticles for the treatment of retinal disorders: Challenges and future prospects
-
Han Z, Conley SM, Naash MI. AAV and compacted DNA nanoparticles for the treatment of retinal disorders: challenges and future prospects. Invest Ophthalmol Vis Sci 2011; 52 : 3051-9.
-
(2011)
Invest Ophthalmol Vis Sci
, vol.52
, pp. 3051-3059
-
-
Han, Z.1
Conley, S.M.2
Naash, M.I.3
-
10
-
-
0028280660
-
Adenovirus vector-mediated in vivo gene transfer into adult murine retina
-
Bennett J, Wilson J, Sun D, et al. Adenovirus vector-mediated in vivo gene transfer into adult murine retina. Invest Ophthalmol Vis Sci 1994; 35 : 2535-42.
-
(1994)
Invest Ophthalmol Vis Sci
, vol.35
, pp. 2535-2542
-
-
Bennett, J.1
Wilson, J.2
Sun, D.3
-
11
-
-
0031687272
-
Immune responses limit adenovirally mediated gene expression in the adult mouse eye
-
Reichel MB, Ali RR, Thrasher AJ, et al. Immune responses limit adenovirally mediated gene expression in the adult mouse eye. Gene Ther 1998; 5 : 1038-46.
-
(1998)
Gene Ther
, vol.5
, pp. 1038-1046
-
-
Reichel, M.B.1
Ali, R.R.2
Thrasher, A.J.3
-
12
-
-
0035940504
-
Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk
-
Vollrath D, Feng W, Duncan JL, et al. Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc Natl Acad Sci USA 2001; 98 : 12584-89.
-
(2001)
Proc Natl Acad Sci USA
, vol.98
, pp. 12584-12589
-
-
Vollrath, D.1
Feng, W.2
Duncan, J.L.3
-
13
-
-
0033582216
-
Adenovirus-mediated regulable target gene expression in vivo
-
Burcin MM, Schiedner G, Kochanek S, et al. Adenovirus-mediated regulable target gene expression in vivo. Proc Natl Acad Sci USA 1999; 96 : 355-60.
-
(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 355-360
-
-
Burcin, M.M.1
Schiedner, G.2
Kochanek, S.3
-
14
-
-
84856966481
-
Ocular gene delivery using lentiviral vectors
-
Balaggan KS, Ali RR. Ocular gene delivery using lentiviral vectors. Gene Ther 2012; 19 : 145-53.
-
(2012)
Gene Ther
, vol.19
, pp. 145-153
-
-
Balaggan, K.S.1
Ali, R.R.2
-
15
-
-
0036797278
-
Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat
-
Duisit G, Conrath H, Saleun S, et al. Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction of retinal pigmented epithelium in rat. Mol Ther 2002; 6 : 446-54.
-
(2002)
Mol Ther
, vol.6
, pp. 446-454
-
-
Duisit, G.1
Conrath, H.2
Saleun, S.3
-
16
-
-
58849087072
-
Equine infectious anemia viral vectormediated codelivery of endostatin and angiostatin driven by retinal pigmented epithelium-specific VMD2 promoter inhibits choroidal neovascularization
-
Kachi S, Binley K, Yokoi K, et al. Equine infectious anemia viral vectormediated codelivery of endostatin and angiostatin driven by retinal pigmented epithelium-specific VMD2 promoter inhibits choroidal neovascularization. Hum Gene Ther 2009; 20 : 31-9.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 31-39
-
-
Kachi, S.1
Binley, K.2
Yokoi, K.3
-
17
-
-
33750855742
-
Lentiviral gene transfer of Rpe65 rescues survival and function of cones in a mouse model of leber congenital amaurosis
-
Bemelmans AP, Kostic C, Crippa SV, et al. Lentiviral gene transfer of Rpe65 rescues survival and function of cones in a mouse model of leber congenital amaurosis. PLoS Med 2006; 3 : 1892-903.
-
(2006)
PLoS Med
, vol.3
, pp. 1892-1903
-
-
Bemelmans, A.P.1
Kostic, C.2
Crippa, S.V.3
-
18
-
-
33645239884
-
Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors
-
Balaggan KS, Binley K, Esapa M, et al. Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors. J Gene Med 2006; 8 : 275-85.
-
(2006)
J Gene Med
, vol.8
, pp. 275-285
-
-
Balaggan, K.S.1
Binley, K.2
Esapa, M.3
-
19
-
-
84892868349
-
Venezuelan equine encephalitis virus-derived glycoprotein-pseudotyped lentivirus vectors differentially transduce corneal endothelium, trabecular meshwork
-
Lipinski D, Barnard A. Venezuelan equine encephalitis virus-derived glycoprotein-pseudotyped lentivirus vectors differentially transduce corneal endothelium, trabecular meshwork. Hum Gene Ther 2013; 25 : 50-62.
-
(2013)
Hum Gene Ther
, vol.25
, pp. 50-62
-
-
Lipinski, D.1
Barnard, A.2
-
20
-
-
84878956736
-
Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: Safety and biodistribution of StarGen for Stargardt disease
-
Binley K, Widdowson P, Loader J, et al. Transduction of photoreceptors with equine infectious anemia virus lentiviral vectors: safety and biodistribution of StarGen for Stargardt disease. Invest Ophthalmol Vis Sci 2013; 54 : 4061-71.
-
(2013)
Invest Ophthalmol Vis Sci
, vol.54
, pp. 4061-4071
-
-
Binley, K.1
Widdowson, P.2
Loader, J.3
-
21
-
-
64549110877
-
Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection
-
Hellström M, Ruitenberg MJ, Pollett MA, et al. Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection. Gene Ther 2009; 16 : 521-32.
-
(2009)
Gene Ther
, vol.16
, pp. 521-532
-
-
Hellström, M.1
Ruitenberg, M.J.2
Pollett, M.A.3
-
22
-
-
35148862553
-
Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors
-
Allocca M, Mussolino C, Garcia-Hoyos M, et al. Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors. J Virol 2007; 81 : 11372-80.
-
(2007)
J Virol
, vol.81
, pp. 11372-11380
-
-
Allocca, M.1
Mussolino, C.2
Garcia-Hoyos, M.3
-
23
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
-
Nathwani AC, Tuddenham EGD, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med 2011; 365 : 2357-65.
-
(2011)
N Engl J Med
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.D.2
Rangarajan, S.3
-
24
-
-
0034050904
-
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
-
Kay MA, Manno CS, Ragni MV, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 2000; 24 : 257-61.
-
(2000)
Nat Genet
, vol.24
, pp. 257-261
-
-
Kay, M.A.1
Manno, C.S.2
Ragni, M.V.3
-
25
-
-
48949120141
-
Recent developments in adeno-associated virus vector technology
-
Büning H, Perabo L, Coutelle O, et al. Recent developments in adeno-associated virus vector technology. J Gene Med 2008; 10 : 717-33.
-
(2008)
J Gene Med
, vol.10
, pp. 717-733
-
-
Büning, H.1
Perabo, L.2
Coutelle, O.3
-
26
-
-
84861998784
-
Intracellular transport of recombinant adeno-associated virus vectors
-
Nonnenmacher M, Weber T. Intracellular transport of recombinant adeno-associated virus vectors. Gene Ther 2012; 19 : 649-58.
-
(2012)
Gene Ther
, vol.19
, pp. 649-658
-
-
Nonnenmacher, M.1
Weber, T.2
-
28
-
-
84861998784
-
Intracellular transport of recombinant adeno-associated virus vectors
-
Nonnenmacher M, Weber T. Intracellular transport of recombinant adeno-associated virus vectors. Gene Ther 2012; 19 : 649-58.
-
(2012)
Gene Ther
, vol.19
, pp. 649-658
-
-
Nonnenmacher, M.1
Weber, T.2
-
29
-
-
45549090635
-
Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
-
Zhong L, Li B, Mah CS, et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci USA 2008; 105 : 7827-32.
-
(2008)
Proc Natl Acad Sci USA
, vol.105
, pp. 7827-7832
-
-
Zhong, L.1
Li, B.2
Mah, C.S.3
-
30
-
-
84876860169
-
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors
-
Kay CN, Ryals RC, Aslanidi GV, et al. Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. PLoS One 2013; 8 : e62097.
-
(2013)
PLoS One
, vol.8
-
-
Kay, C.N.1
Ryals, R.C.2
Aslanidi, G.V.3
-
31
-
-
79551634862
-
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina
-
Petrs-Silva H, Dinculescu A, Li Q, et al. Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol Ther 2011; 19 : 293-301.
-
(2011)
Mol Ther
, vol.19
, pp. 293-301
-
-
Petrs-Silva, H.1
Dinculescu, A.2
Li, Q.3
-
32
-
-
84880540708
-
In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous
-
Dalkara D, Byrne LLC, Klimczak RR, et al. In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous. Sci Transl Med 2013 ; 5 :189ra76.
-
(2013)
Sci Transl Med
, vol.5
-
-
Dalkara, D.1
Byrne, L.L.C.2
Klimczak, R.R.3
-
33
-
-
32344438754
-
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
-
Maheshri N, Koerber JT, Kaspar BK, Schaffer DV. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 2006; 24 : 198-204.
-
(2006)
Nat Biotechnol
, vol.24
, pp. 198-204
-
-
Maheshri, N.1
Koerber, J.T.2
Kaspar, B.K.3
Schaffer, D.V.4
-
34
-
-
0042360384
-
Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors
-
Müller OJ, Kaul F, Weitzman MD, et al. Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat Biotechnol 2003; 21 : 1040-6.
-
(2003)
Nat Biotechnol
, vol.21
, pp. 1040-1046
-
-
Müller, O.J.1
Kaul, F.2
Weitzman, M.D.3
-
35
-
-
70449338146
-
A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Müller cells
-
Klimczak RR, Koerber JT, Dalkara D, et al. A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Müller cells. PLoS One 2009; 4 : e7467.
-
(2009)
PLoS One
, vol.4
-
-
Klimczak, R.R.1
Koerber, J.T.2
Dalkara, D.3
-
36
-
-
80052401649
-
AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa
-
Dalkara D, Kolstad KD, Guerin KI, et al. AAV mediated GDNF secretion from retinal glia slows down retinal degeneration in a rat model of retinitis pigmentosa. Mol Ther 2011; 19 : 1602-8.
-
(2011)
Mol Ther
, vol.19
, pp. 1602-1608
-
-
Dalkara, D.1
Kolstad, K.D.2
Guerin, K.I.3
-
37
-
-
79956006692
-
Intravitreal injection of AAV2 transduces macaque inner retina
-
Yin L, Greenberg K, Hunter JJ, et al. Intravitreal injection of AAV2 transduces macaque inner retina. Invest Ophthalmol Vis Sci 2011; 52 : 2775-83.
-
(2011)
Invest Ophthalmol Vis Sci
, vol.52
, pp. 2775-2783
-
-
Yin, L.1
Greenberg, K.2
Hunter, J.J.3
-
38
-
-
73849132937
-
Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous
-
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.
-
(2009)
Mol Ther
, vol.17
, pp. 2096-2102
-
-
Dalkara, D.1
Kolstad, K.D.2
Caporale, N.3
-
39
-
-
61649095399
-
High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors
-
Petrs-Silva H, Dinculescu A, Li Q, et al. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Mol Ther 2009; 17 : 463-71.
-
(2009)
Mol Ther
, vol.17
, pp. 463-471
-
-
Petrs-Silva, H.1
Dinculescu, A.2
Li, Q.3
-
40
-
-
85027946862
-
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness
-
Doroudchi MM, Greenberg KP, Liu J, et al. Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness. Mol Ther 2011; 19 : 1220-9.
-
(2011)
Mol Ther
, vol.19
, pp. 1220-1229
-
-
Doroudchi, M.M.1
Greenberg, K.P.2
Liu, J.3
-
41
-
-
67650802500
-
Antioxidant or neurotrophic factor treatment preserves function in a mouse model of neovascularizationassociated oxidative stress
-
Dorrell MI, Aguilar E, Jacobson R, et al. Antioxidant or neurotrophic factor treatment preserves function in a mouse model of neovascularizationassociated oxidative stress. J Clin Invest 2009; 119 : 611-23.
-
(2009)
J Clin Invest
, vol.119
, pp. 611-623
-
-
Dorrell, M.I.1
Aguilar, E.2
Jacobson, R.3
-
42
-
-
73849091644
-
Molecular evolution of adenoassociated virus for enhanced glial gene delivery
-
Koerber JT, Klimczak R, Jang JH, et al. Molecular evolution of adenoassociated virus for enhanced glial gene delivery. Mol Ther 2009; 17 : 2088-95.
-
(2009)
Mol Ther
, vol.17
, pp. 2088-2095
-
-
Koerber, J.T.1
Klimczak, R.2
Jang, J.H.3
-
43
-
-
0035894739
-
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: The retina as a model
-
Auricchio A, Kobinger G, Anand V, et al. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum Mol Genet 2001; 10 : 3075-81.
-
(2001)
Hum Mol Genet
, vol.10
, pp. 3075-3081
-
-
Auricchio, A.1
Kobinger, G.2
Anand, V.3
-
44
-
-
79551634862
-
Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina
-
Petrs-Silva H, Dinculescu A, Li Q, et al. Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Mol Ther 2009; 19 : 293-301.
-
(2009)
Mol Ther
, vol.19
, pp. 293-301
-
-
Petrs-Silva, H.1
Dinculescu, A.2
Li, Q.3
-
45
-
-
40249098715
-
Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8
-
Natkunarajah M, Trittibach P, McIntosh J, et al. Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Ther 2008; 15 : 463-7.
-
(2008)
Gene Ther
, vol.15
, pp. 463-467
-
-
Natkunarajah, M.1
Trittibach, P.2
McIntosh, J.3
-
46
-
-
10744229842
-
Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery
-
Weber M, Rabinowitz J, Provost N, et al. Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery. Mol Ther 2003; 7 : 774-81.
-
(2003)
Mol Ther
, vol.7
, pp. 774-781
-
-
Weber, M.1
Rabinowitz, J.2
Provost, N.3
-
47
-
-
43049119982
-
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
-
Allocca M, Doria M, Petrillo M, et al. Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest 2008; 118 : 1955-64.
-
(2008)
J Clin Invest
, vol.118
, pp. 1955-1964
-
-
Allocca, M.1
Doria, M.2
Petrillo, M.3
-
48
-
-
13044256410
-
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina
-
Bennett J, Maguire AM, Cideciyan AV, et al. Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina. Proc Natl Acad Sci USA 1999; 96 : 9920-5.
-
(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 9920-9925
-
-
Bennett, J.1
Maguire, A.M.2
Cideciyan, A.V.3
-
49
-
-
84873830812
-
AAV9 targets cone photoreceptors in the nonhuman primate retina
-
Vandenberghe LH, Bell P, Maguire AM, et al. AAV9 targets cone photoreceptors in the nonhuman primate retina. PLoS One 2013; 8 : e53463.
-
(2013)
PLoS One
, vol.8
-
-
Vandenberghe, L.H.1
Bell, P.2
Maguire, A.M.3
-
50
-
-
79956006692
-
Intravitreal injection of AAV2 transduces macaque inner retina
-
Yin L, Greenberg K, Hunter JJ, et al. Intravitreal injection of AAV2 transduces macaque inner retina. Invest Ophthalmol Vis Sci 2011; 52 : 2775-83.
-
(2011)
Invest Ophthalmol Vis Sci
, vol.52
, pp. 2775-2783
-
-
Yin, L.1
Greenberg, K.2
Hunter, J.J.3
|