-
1
-
-
0031011667
-
Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus
-
J. G. Flannery, S. Zolotukhin, M. I. Vaquero, J. M. LaVail, N. Muzyczka, and W. W. Hauswirth, "Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus," Proc. Natl. Acad. Sci. U.S.A. 94, 6916-6921 (1997).
-
(1997)
Proc. Natl. Acad. Sci. U.S.A
, vol.94
, pp. 6916-6921
-
-
Flannery, J.G.1
Zolotukhin, S.2
Vaquero, M.I.3
LaVail, J.M.4
Muzyczka, N.5
Hauswirth, W.W.6
-
2
-
-
13044256410
-
Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina
-
J. Bennett, A. M. Maguire, V Cideciyan, M. Schnell, E. Glover, V. Anand, T. S. Aleman, N. Chirmule, A. R. Gupta, Y. Huang, G.-P. Gao, W. C. Nyberg, J. Tazelaar, J. Hughes, J. M. Wilson, and S. G. Jacobson, "Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina," Proc. Natl. Acad. Sci. U.S.A. 96, 9920-9925 (1999).
-
(1999)
Proc. Natl. Acad. Sci. U.S.A
, vol.96
, pp. 9920-9925
-
-
Bennett, J.1
Maguire, A.M.2
Cideciyan, V.3
Schnell, M.4
Glover, E.5
Anand, V.6
Aleman, T.S.7
Chirmule, N.8
Gupta, A.R.9
Huang, Y.10
Gao, G.-P.11
Nyberg, W.C.12
Tazelaar, J.13
Hughes, J.14
Wilson, J.M.15
Jacobson, S.G.16
-
3
-
-
0033106311
-
Evaluation of adeno-associated virus-mediated gene transfer into the rat retina by clincal fluorescence photography
-
F. Rolling, W. Shen, H. Tabarias, I. Constable, Y. Kanagasingam, C. J. Barry, and P. E. Rakoczy, "Evaluation of adeno-associated virus-mediated gene transfer into the rat retina by clincal fluorescence photography," Hum. Gene Ther. 10, 641-648 (1999).
-
(1999)
Hum. Gene Ther
, vol.10
, pp. 641-648
-
-
Rolling, F.1
Shen, W.2
Tabarias, H.3
Constable, I.4
Kanagasingam, Y.5
Barry, C.J.6
Rakoczy, P.E.7
-
4
-
-
0034058476
-
Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction
-
J. Bennett, V. Anand, G. M. Acland, and A. M. Maguire, "Cross-species comparison of in vivo reporter gene expression after recombinant adeno-associated virus-mediated retinal transduction," Methods Enzymol. 316, 777-789 (2000).
-
(2000)
Methods Enzymol
, vol.316
, pp. 777-789
-
-
Bennett, J.1
Anand, V.2
Acland, G.M.3
Maguire, A.M.4
-
5
-
-
0035032662
-
Gene therapy restores vision in a canine model of childhood blindness
-
G. M. Acland, G. D. Aguirre, J. Ray, Q. Zhang, T. S. Aleman, A. V. Cideciyan, S. E. Pearce-Kelling, V. Anand, Y. Zeng, A. M. Maguire, S. G. Jacobson, W. W. Hauswirth, and J. Bennett, "Gene therapy restores vision in a canine model of childhood blindness," Nat. Genet. 28, 92-95 (2001).
-
(2001)
Nat. Genet
, vol.28
, pp. 92-95
-
-
Acland, G.M.1
Aguirre, G.D.2
Ray, J.3
Zhang, Q.4
Aleman, T.S.5
Cideciyan, A.V.6
Pearce-Kelling, S.E.7
Anand, V.8
Zeng, Y.9
Maguire, A.M.10
Jacobson, S.G.11
Hauswirth, W.W.12
Bennett, J.13
-
6
-
-
0036140216
-
Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity
-
J. E. Rabinowitz, F. Rolling, C. Li, H. Conrath, W. Xiao, X. Xiao, and R. J. Samulski, "Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity," J. Virol. 76, 791-801 (2002).
-
(2002)
J. Virol
, vol.76
, pp. 791-801
-
-
Rabinowitz, J.E.1
Rolling, F.2
Li, C.3
Conrath, H.4
Xiao, W.5
Xiao, X.6
Samulski, R.J.7
-
7
-
-
0036180817
-
Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus
-
G. M. Sarra, C. Stephens, F. C. Schlichtenbrede, J. W. Bainbridge, A. J. Thrasher, P. J. Luthert, and R. R. Ali, "Kinetics of transgene expression in mouse retina following sub-retinal injection of recombinant adeno-associated virus," Vision Res. 42, 541-549 (2002).
-
(2002)
Vision Res
, vol.42
, pp. 541-549
-
-
Sarra, G.M.1
Stephens, C.2
Schlichtenbrede, F.C.3
Bainbridge, J.W.4
Thrasher, A.J.5
Luthert, P.J.6
Ali, R.R.7
-
8
-
-
0036310297
-
Virus-mediated transduction of murine retina with adeno-associated virus: Effects of viral capsid and genome size
-
G. S. Yang, M. Schmidt, Z. Yan, J. D. Lindbloom, T. C. Harding, B. A. Donahue, J. F. Engelhardt, R. Kotin, and B. L. Davidson, "Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size," J. Virol. 76, 7651-7660 (2002).
-
(2002)
J. Virol
, vol.76
, pp. 7651-7660
-
-
Yang, G.S.1
Schmidt, M.2
Yan, Z.3
Lindbloom, J.D.4
Harding, T.C.5
Donahue, B.A.6
Engelhardt, J.F.7
Kotin, R.8
Davidson, B.L.9
-
9
-
-
0344468070
-
Quantitative model demonstrating that recombinant adeno-associated virus and green fluorescent protein are non-toxic to the rat retina
-
D. M. Daniels, W. Y. Shen, I. J. Constable, and P. E. Rakoczy, "Quantitative model demonstrating that recombinant adeno-associated virus and green fluorescent protein are non-toxic to the rat retina," Graefe's Arch. Clin. Exp. Ophthalmol. 31, 439-444 (2003).
-
(2003)
Graefe's Arch. Clin. Exp. Ophthalmol
, vol.31
, pp. 439-444
-
-
Daniels, D.M.1
Shen, W.Y.2
Constable, I.J.3
Rakoczy, P.E.4
-
10
-
-
10744225428
-
Adeno-associated virus type 5: Transduction efficiency and cell-type specificity in the primate retina
-
A. J. Lotery, G. S. Yang, R. F. Mullins, S. R. Russell, M. Schmidt, E. M. Stone, J. D. Lindbloom, J. A. Chiorini, R. M. Kotin, and B. L. Davidson, "Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina," Hum. Gene Ther. 14, 1663-1671 (2003).
-
(2003)
Hum. Gene Ther
, vol.14
, pp. 1663-1671
-
-
Lotery, A.J.1
Yang, G.S.2
Mullins, R.F.3
Russell, S.R.4
Schmidt, M.5
Stone, E.M.6
Lindbloom, J.D.7
Chiorini, J.A.8
Kotin, R.M.9
Davidson, B.L.10
-
11
-
-
0344716689
-
Practical considerations of recombinant adeno-associated virus-mediated gene transfer for treatment of retinal degenerations
-
W. Y. Shen, C. M. Lai, Y. K. Lai, D. Zhang, T. Zaknich, E. N. Sutanto, I. J. Constable, and P. E. Rakoczy, "Practical considerations of recombinant adeno-associated virus-mediated gene transfer for treatment of retinal degenerations," J. Gene Med. 5, 576-587 (2003).
-
(2003)
J. Gene Med
, vol.5
, pp. 576-587
-
-
Shen, W.Y.1
Lai, C.M.2
Lai, Y.K.3
Zhang, D.4
Zaknich, T.5
Sutanto, E.N.6
Constable, I.J.7
Rakoczy, P.E.8
-
12
-
-
28444442243
-
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
-
G. M. Acland, G. D. Aguirre, J. Bennett, T. S. Aleman, A. V. Cideciyan, J. Bennicelli, N. S. Dejneka, S. E. Pearce-Kelling, A. M. Maguire, K. Palczewski, W. W. Hauswirth, and S. G. Jacobson, "Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness," Mol. Therapy 12, 1072-1082 (2005).
-
(2005)
Mol. Therapy
, vol.12
, pp. 1072-1082
-
-
Acland, G.M.1
Aguirre, G.D.2
Bennett, J.3
Aleman, T.S.4
Cideciyan, A.V.5
Bennicelli, J.6
Dejneka, N.S.7
Pearce-Kelling, S.E.8
Maguire, A.M.9
Palczewski, K.10
Hauswirth, W.W.11
Jacobson, S.G.12
-
13
-
-
25144501446
-
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis
-
S. H. Min, L. L. Molday, M. W. Seeliger, A. Dinculesco, A. M. Timmers, A. Janssen, F. Tonagel, N. Tanimoto, B. H. Weber, R. S. Molday, and W. W. Hauswirth, "Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis," Mol. Therapy 12, 644-651 (2005).
-
(2005)
Mol. Therapy
, vol.12
, pp. 644-651
-
-
Min, S.H.1
Molday, L.L.2
Seeliger, M.W.3
Dinculesco, A.4
Timmers, A.M.5
Janssen, A.6
Tonagel, F.7
Tanimoto, N.8
Weber, B.H.9
Molday, R.S.10
Hauswirth, W.W.11
-
14
-
-
33748106526
-
Human blue-opsin promoter preferentially targets reporter gene expression to rat S-cone photoreceptors
-
L. G. Glushakova, A. M. Timmers, J. Pang, J. T. Teusner, and W. W. Hauswirth, "Human blue-opsin promoter preferentially targets reporter gene expression to rat S-cone photoreceptors," Invest. Ophthalmol. Visual Sci. 47, 3505-3513(2006).
-
(2006)
Invest. Ophthalmol. Visual Sci
, vol.47
, pp. 3505-3513
-
-
Glushakova, L.G.1
Timmers, A.M.2
Pang, J.3
Teusner, J.T.4
Hauswirth, W.W.5
-
15
-
-
32944473999
-
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis
-
J. J. Pang, B. Chang, A. Kumar, S. Nusinowitz, S. M. Noorwez, J. Li, A. Rani, T. C. Foster, V. A. Chiodo, T. Doyle, H. Li, R. Malhotra, J. T. Teusner, J. H. McDowell, S. H. Min, Q. Li, S. Kaushal, and W. W. Hauswirth, "Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis," Mol. Therapy 13, 565-572 (2006).
-
(2006)
Mol. Therapy
, vol.13
, pp. 565-572
-
-
Pang, J.J.1
Chang, B.2
Kumar, A.3
Nusinowitz, S.4
Noorwez, S.M.5
Li, J.6
Rani, A.7
Foster, T.C.8
Chiodo, V.A.9
Doyle, T.10
Li, H.11
Malhotra, R.12
Teusner, J.T.13
McDowell, J.H.14
Min, S.H.15
Li, Q.16
Kaushal, S.17
Hauswirth, W.W.18
-
16
-
-
0002291134
-
Genetics and biology of adeno-associated virus
-
M. G. Kaplitt and A. D. Loewy, eds, Academic
-
J. S. Bartlett and R. J. Samulski, "Genetics and biology of adeno-associated virus," in Viral Vectors: Gene Therapy and Neuroscience Applications, M. G. Kaplitt and A. D. Loewy, eds. (Academic, 1995), pp. 55-73.
-
(1995)
Viral Vectors: Gene Therapy and Neuroscience Applications
, pp. 55-73
-
-
Bartlett, J.S.1
Samulski, R.J.2
-
17
-
-
0034724209
-
Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
-
B. L. Davidson, C. S. Stein, J. A. Heth, I. Martins, R. M. Kotin, T. A. Derksen, J. Zabner, A. Ghodsi, and J. A. Chiorini, "Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system," Proc. Natl. Acad. Sci. U.S.A. 97, 3428-3432 (2000).
-
(2000)
Proc. Natl. Acad. Sci. U.S.A
, vol.97
, pp. 3428-3432
-
-
Davidson, B.L.1
Stein, C.S.2
Heth, J.A.3
Martins, I.4
Kotin, R.M.5
Derksen, T.A.6
Zabner, J.7
Ghodsi, A.8
Chiorini, J.A.9
-
18
-
-
16844385431
-
Postsurgical assessment and long-term safety of recombinant adeno-associated virus-mediated gene transfer into the retinas of dogs and primates
-
G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, R Moullier, and F. Rolling, "Postsurgical assessment and long-term safety of recombinant adeno-associated virus-mediated gene transfer into the retinas of dogs and primates," Arch. Ophthalmol. (Chicago) 123, 500-506 (2005).
-
(2005)
Arch. Ophthalmol. (Chicago)
, vol.123
, pp. 500-506
-
-
LeMeur, G.1
Weber, M.2
Yann, P.3
Mendes-Madeira, A.4
Nivard, D.5
Deschamps, J.6
Moullier, R.7
Rolling, F.8
-
19
-
-
33744495152
-
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
-
S. G. Jacobson, G. M. Acland, G. D. Aguirre, T. S. Aleman, S. B. Schwartz, A. V. Cideciyan, C. J. Zeiss, A. M. Komaromy, S. Kaushal, A. J. Roman, E. A. Windsor, A. Sumaroka, S. E. Pearce-Kelling, T. J. Conlon, V. A. Chiodo, S. L. Boye, T. R. Flotte, A. M. Maguire, J. Bennett, and W. W. Hauswirth, "Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection," Mol. Therapy 13, 1074-1084 (2006).
-
(2006)
Mol. Therapy
, vol.13
, pp. 1074-1084
-
-
Jacobson, S.G.1
Acland, G.M.2
Aguirre, G.D.3
Aleman, T.S.4
Schwartz, S.B.5
Cideciyan, A.V.6
Zeiss, C.J.7
Komaromy, A.M.8
Kaushal, S.9
Roman, A.J.10
Windsor, E.A.11
Sumaroka, A.12
Pearce-Kelling, S.E.13
Conlon, T.J.14
Chiodo, V.A.15
Boye, S.L.16
Flotte, T.R.17
Maguire, A.M.18
Bennett, J.19
Hauswirth, W.W.20
more..
-
20
-
-
33750591238
-
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber Congenital Amaurosis
-
S. G. Jacobson, S. L. Boye, T. S. Aleman, T. J. Conlon, C. J. Zeiss, A. J. Roman, A. V. Cideciyan, S. B. Schwartz, A. M. Komaromy, M. Doobrajh, A. Y. Cheung, A. Sumaroka, S. E. Pearce-Kelling, G. D. Aguirre, S. Kaushal, A. M. Maguire, T. R. Flotte, and W. W. Hauswirth, "Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber Congenital Amaurosis," Hum. Gene Ther. 17, 845-858 (2006).
-
(2006)
Hum. Gene Ther
, vol.17
, pp. 845-858
-
-
Jacobson, S.G.1
Boye, S.L.2
Aleman, T.S.3
Conlon, T.J.4
Zeiss, C.J.5
Roman, A.J.6
Cideciyan, A.V.7
Schwartz, S.B.8
Komaromy, A.M.9
Doobrajh, M.10
Cheung, A.Y.11
Sumaroka, A.12
Pearce-Kelling, S.E.13
Aguirre, G.D.14
Kaushal, S.15
Maguire, A.M.16
Flotte, T.R.17
Hauswirth, W.W.18
-
21
-
-
34250758909
-
Restoration of cone function in a mouse model of achromatopsia by rAAV mediated promoter targeted expression of cone specific alpha-transducin
-
presented at the, Fort Lauderdale, Florida, May 1-5
-
J. J. Alexander, Q. Li, A. M. Timmers, N. L. Hawes, B. Chang, and W. W. Hauswirth, "Restoration of cone function in a mouse model of achromatopsia by rAAV mediated promoter targeted expression of cone specific alpha-transducin," presented at the Association for Research in Vision and Ophthalmology (ARVO), Annual Meeting, Fort Lauderdale, Florida, May 1-5, 2005.
-
(2005)
Association for Research in Vision and Ophthalmology (ARVO), Annual Meeting
-
-
Alexander, J.J.1
Li, Q.2
Timmers, A.M.3
Hawes, N.L.4
Chang, B.5
Hauswirth, W.W.6
-
22
-
-
0038253683
-
Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina
-
J. W. Bainbridge, A. Mistry, F. C. Schlichtenbrede, A. Smith, C. Broderick, M. De Alwis, A. Georgiadis, P. M. Taylor, M. Squires, C. Sethi, D. Charteris, A. J. Thrasher, D. Sargan, and R. R. Ali, "Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina," Gene Ther. 10, 1336-1344 (2003).
-
(2003)
Gene Ther
, vol.10
, pp. 1336-1344
-
-
Bainbridge, J.W.1
Mistry, A.2
Schlichtenbrede, F.C.3
Smith, A.4
Broderick, C.5
De Alwis, M.6
Georgiadis, A.7
Taylor, P.M.8
Squires, M.9
Sethi, C.10
Charteris, D.11
Thrasher, A.J.12
Sargan, D.13
Ali, R.R.14
-
23
-
-
0029995531
-
A humanized green fluorescent protein cDNA adapted for high-level expression in mammalian cells
-
S. Zolotukhin, M. Potter, W. W. Hauswirth, J. Guy, and N. Muzyczka, "A humanized green fluorescent protein cDNA adapted for high-level expression in mammalian cells," J. Virol. 70, 4646-4654 (1996).
-
(1996)
J. Virol
, vol.70
, pp. 4646-4654
-
-
Zolotukhin, S.1
Potter, M.2
Hauswirth, W.W.3
Guy, J.4
Muzyczka, N.5
-
24
-
-
0026806222
-
A locus control region adjacent to the human red and green visual pigment genes
-
Y. Wang, J. P. Macke, S. L. Merbs, D. J. Zack, B. Klaunberg, J. Bennett, J. Gearhart, and J. Nathans, "A locus control region adjacent to the human red and green visual pigment genes," Neuron 9, 429-440 (1992).
-
(1992)
Neuron
, vol.9
, pp. 429-440
-
-
Wang, Y.1
Macke, J.P.2
Merbs, S.L.3
Zack, D.J.4
Klaunberg, B.5
Bennett, J.6
Gearhart, J.7
Nathans, J.8
-
25
-
-
0022696951
-
Molecular genetics of inherited variation in human color vision
-
J. Nathans, T. P. Piantanida, R. L. Eddy, T. B. Shows, and D. S. Hogness, "Molecular genetics of inherited variation in human color vision," Science 232, 203-210 (1986).
-
(1986)
Science
, vol.232
, pp. 203-210
-
-
Nathans, J.1
Piantanida, T.P.2
Eddy, R.L.3
Shows, T.B.4
Hogness, D.S.5
-
26
-
-
0019458230
-
The topography of primate retina: A study of the human, bushbaby, and new- and old-world monkeys
-
J. Stone and E. Johnston, "The topography of primate retina: a study of the human, bushbaby, and new- and old-world monkeys," J. Comp. Neurol. 196, 205-223 (1981).
-
(1981)
J. Comp. Neurol
, vol.196
, pp. 205-223
-
-
Stone, J.1
Johnston, E.2
-
27
-
-
0034469055
-
Conservation of absolute foveal area in New World Monkeys
-
E. C. S. Franco, B. L. Finlay, L. C. L. Silveira, E. S. Yamada, and J. C. Crowley, "Conservation of absolute foveal area in New World Monkeys," Br. Behavior Evol. 56, 276-286(2000).
-
(2000)
Br. Behavior Evol
, vol.56
, pp. 276-286
-
-
Franco, E.C.S.1
Finlay, B.L.2
Silveira, L.C.L.3
Yamada, E.S.4
Crowley, J.C.5
-
28
-
-
0034146816
-
Spatial order in short-wavelength-sensitive cone photoreceptors: A comparative study of the primate retina
-
P. R. Martin, U. Grunert, T. L. Chan, and K. Bumsted, "Spatial order in short-wavelength-sensitive cone photoreceptors: A comparative study of the primate retina," J. Opt. Soc. Am. 17, 557-579 (2000).
-
(2000)
J. Opt. Soc. Am
, vol.17
, pp. 557-579
-
-
Martin, P.R.1
Grunert, U.2
Chan, T.L.3
Bumsted, K.4
-
29
-
-
0036797278
-
Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction. of retinal pigmented epithelium in rat
-
G. Duisit, H. Conrath, S. Saleun, S. Folliot, N. Provost, F. Cosset, V. Sandrin, P. Moullier, and F. Rolling, "Five recombinant simian immunodeficiency virus pseudotypes lead to exclusive transduction. of retinal pigmented epithelium in rat," Mol. Therapy 6, 446-454 (2002).
-
(2002)
Mol. Therapy
, vol.6
, pp. 446-454
-
-
Duisit, G.1
Conrath, H.2
Saleun, S.3
Folliot, S.4
Provost, N.5
Cosset, F.6
Sandrin, V.7
Moullier, P.8
Rolling, F.9
-
30
-
-
33747421037
-
Gene delivery to the retina using lentiviral vectors
-
J. G. Hollyfield, R. E. Anderson, and M. M. LaVail, eds, Springer
-
K. P. Greenberg, E. S. Lee, D. V. Schaffer, and J. G. Flannery, "Gene delivery to the retina using lentiviral vectors," in Retinal and Degenerative Diseases (Advances in Experimental Medicine and Biology), J. G. Hollyfield, R. E. Anderson, and M. M. LaVail, eds. (Springer, 2006), pp. 255-266.
-
(2006)
Retinal and Degenerative Diseases (Advances in Experimental Medicine and Biology)
, pp. 255-266
-
-
Greenberg, K.P.1
Lee, E.S.2
Schaffer, D.V.3
Flannery, J.G.4
|