Abstract

Recombinant adeno-associated virus (rAAV) is a promising vector for gene therapy of photoreceptor-based diseases. Previous studies have demonstrated that rAAV serotypes 2 and 5 can transduce both rod and cone photoreceptors in rodents and dogs, and it can target rods, but not cones in primates. Here we report that using a human cone-specific enhancer and promoter to regulate expression of a green fluorescent protein (GFP) reporter gene in an rAAV-5 vector successfully targeted expression of the reporter gene to primate cones, and the time course of GFP expression was able to be monitored in a living animal using the RetCam II digital imaging system.

© 2007 Optical Society of America

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    [CrossRef] [PubMed]
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    [CrossRef] [PubMed]
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    [CrossRef] [PubMed]
  4. 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).
    [CrossRef] [PubMed]
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    [CrossRef] [PubMed]
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    [CrossRef]
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    [CrossRef] [PubMed]
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    [PubMed]
  24. 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).
    [CrossRef] [PubMed]
  25. 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).
    [CrossRef] [PubMed]
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    [CrossRef] [PubMed]
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    [CrossRef]
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    [CrossRef]
  29. 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).
    [CrossRef]
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    [CrossRef]

2006 (4)

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

2005 (3)

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).
[CrossRef]

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).
[CrossRef]

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

2003 (4)

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).
[CrossRef] [PubMed]

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).

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

2002 (4)

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

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).
[CrossRef]

2001 (1)

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).
[CrossRef] [PubMed]

2000 (4)

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

1999 (2)

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

1997 (1)

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).
[CrossRef] [PubMed]

1996 (1)

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).
[PubMed]

1992 (1)

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).
[CrossRef] [PubMed]

1986 (1)

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).
[CrossRef] [PubMed]

1981 (1)

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).
[CrossRef] [PubMed]

Acland, G. M.

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Aguirre, G. D.

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Aleman, T. S.

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).
[CrossRef] [PubMed]

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).
[CrossRef]

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).
[CrossRef]

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).
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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).
[CrossRef] [PubMed]

Alexander, J. J.

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.

Ali, R. R.

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).
[CrossRef] [PubMed]

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).
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Anand, V.

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).
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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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Bainbridge, J. W.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Barry, C. J.

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).
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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.

Bennett, J.

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

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).
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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).
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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).
[CrossRef]

Boye, S. L.

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Broderick, C.

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).
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Bumsted, K.

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).
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Chan, T. L.

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).
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Chang, B.

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).
[CrossRef]

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.

Charteris, D.

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).
[CrossRef] [PubMed]

Cheung, A. Y.

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).
[CrossRef] [PubMed]

Chiodo, V. A.

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).
[CrossRef]

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).
[CrossRef]

Chiorini, J. A.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Chirmule, N.

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).
[CrossRef] [PubMed]

Cideciyan, A. V.

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Cideciyan, V.

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).
[CrossRef] [PubMed]

Conlon, T. J.

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).
[CrossRef] [PubMed]

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).
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Conrath, H.

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).
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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).
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Constable, I.

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).
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Constable, I. J.

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).
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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).

Cosset, F.

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).
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Crowley, J. C.

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).
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Daniels, D. M.

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).

Davidson, B. L.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

De Alwis, M.

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).
[CrossRef] [PubMed]

Dejneka, N. S.

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).
[CrossRef]

Derksen, T. A.

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).
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Deschamps, J.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

Dinculesco, A.

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).
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Donahue, B. A.

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).
[CrossRef] [PubMed]

Doobrajh, M.

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).
[CrossRef] [PubMed]

Doyle, T.

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).
[CrossRef]

Duisit, G.

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).
[CrossRef]

Eddy, R. L.

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).
[CrossRef] [PubMed]

Engelhardt, J. F.

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).
[CrossRef] [PubMed]

Finlay, B. L.

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).
[CrossRef]

Flannery, J. G.

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).
[CrossRef] [PubMed]

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.
[CrossRef]

Flotte, T. R.

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).
[CrossRef] [PubMed]

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).
[CrossRef]

Folliot, S.

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).
[CrossRef]

Foster, T. C.

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).
[CrossRef]

Franco, E. C. S.

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).
[CrossRef]

Gao, G.-P.

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).
[CrossRef] [PubMed]

Gearhart, J.

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).
[CrossRef] [PubMed]

Georgiadis, A.

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).
[CrossRef] [PubMed]

Ghodsi, A.

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).
[CrossRef] [PubMed]

Glover, E.

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).
[CrossRef] [PubMed]

Glushakova, L. G.

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).
[CrossRef]

Greenberg, K. P.

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.
[CrossRef]

Grunert, U.

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).
[CrossRef]

Gupta, A. R.

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).
[CrossRef] [PubMed]

Guy, J.

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).
[PubMed]

Harding, T. C.

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).
[CrossRef] [PubMed]

Hauswirth, W. W.

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

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).
[PubMed]

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.

Hawes, N. L.

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.

Heth, J. A.

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).
[CrossRef] [PubMed]

Hogness, D. S.

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).
[CrossRef] [PubMed]

Huang, Y.

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).
[CrossRef] [PubMed]

Hughes, J.

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).
[CrossRef] [PubMed]

Jacobson, S. G.

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Janssen, A.

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).
[CrossRef]

Johnston, E.

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).
[CrossRef] [PubMed]

Kanagasingam, Y.

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).
[CrossRef] [PubMed]

Kaushal, S.

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Klaunberg, B.

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).
[CrossRef] [PubMed]

Komaromy, A. M.

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).
[CrossRef] [PubMed]

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).
[CrossRef]

Kotin, R.

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).
[CrossRef] [PubMed]

Kotin, R. M.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Kumar, A.

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).
[CrossRef]

Lai, C. M.

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).
[CrossRef] [PubMed]

Lai, Y. K.

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).
[CrossRef] [PubMed]

LaVail, J. M.

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).
[CrossRef] [PubMed]

Lee, E. S.

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.
[CrossRef]

LeMeur, G.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

Li, C.

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).
[CrossRef]

Li, H.

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).
[CrossRef]

Li, J.

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).
[CrossRef]

Li, Q.

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).
[CrossRef]

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.

Lindbloom, J. D.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Lotery, A. J.

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).
[CrossRef] [PubMed]

Luthert, P. J.

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).
[CrossRef] [PubMed]

Macke, J. P.

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).
[CrossRef] [PubMed]

Maguire, A. M.

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).
[CrossRef] [PubMed]

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Malhotra, R.

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).
[CrossRef]

Martin, P. R.

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).
[CrossRef]

Martins, I.

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).
[CrossRef] [PubMed]

McDowell, J. H.

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).
[CrossRef]

Mendes-Madeira, A.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

Merbs, S. L.

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).
[CrossRef] [PubMed]

Min, S. H.

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).
[CrossRef]

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).
[CrossRef]

Mistry, A.

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).
[CrossRef] [PubMed]

Molday, L. L.

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).
[CrossRef]

Molday, R. S.

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).
[CrossRef]

Moullier, P.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

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).
[CrossRef]

Mullins, R. F.

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).
[CrossRef] [PubMed]

Muzyczka, N.

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).
[CrossRef] [PubMed]

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).
[PubMed]

Nathans, J.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Nivard, D.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

Noorwez, S. M.

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).
[CrossRef]

Nusinowitz, S.

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).
[CrossRef]

Nyberg, W. C.

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).
[CrossRef] [PubMed]

Palczewski, K.

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).
[CrossRef]

Pang, J.

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).
[CrossRef]

Pang, J. J.

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).
[CrossRef]

Pearce-Kelling, S. E.

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).
[CrossRef]

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).
[CrossRef] [PubMed]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Piantanida, T. P.

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).
[CrossRef] [PubMed]

Potter, M.

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).
[PubMed]

Provost, N.

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).
[CrossRef]

Rabinowitz, J. E.

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).
[CrossRef]

Rakoczy, P. E.

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).
[CrossRef] [PubMed]

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).

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).
[CrossRef] [PubMed]

Rani, A.

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).
[CrossRef]

Ray, J.

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).
[CrossRef] [PubMed]

Rolling, F.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

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).
[CrossRef]

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Roman, A. J.

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Russell, S. R.

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).
[CrossRef] [PubMed]

Saleun, S.

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).
[CrossRef]

Samulski, R. J.

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).
[CrossRef]

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.

Sandrin, V.

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).
[CrossRef]

Sargan, D.

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).
[CrossRef] [PubMed]

Sarra, G. M.

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).
[CrossRef] [PubMed]

Schaffer, D. V.

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.
[CrossRef]

Schlichtenbrede, F. C.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Schmidt, M.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Schnell, M.

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).
[CrossRef] [PubMed]

Schwartz, S. B.

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).
[CrossRef] [PubMed]

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).
[CrossRef]

Seeliger, M. W.

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).
[CrossRef]

Sethi, C.

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).
[CrossRef] [PubMed]

Shen, W.

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).
[CrossRef] [PubMed]

Shen, W. Y.

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).

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).
[CrossRef] [PubMed]

Shows, T. B.

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).
[CrossRef] [PubMed]

Silveira, L. C. L.

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).
[CrossRef]

Smith, A.

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).
[CrossRef] [PubMed]

Squires, M.

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).
[CrossRef] [PubMed]

Stein, C. S.

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).
[CrossRef] [PubMed]

Stephens, C.

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).
[CrossRef] [PubMed]

Stone, E. M.

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).
[CrossRef] [PubMed]

Stone, J.

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).
[CrossRef] [PubMed]

Sumaroka, A.

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).
[CrossRef] [PubMed]

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).
[CrossRef]

Sutanto, E. N.

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).
[CrossRef] [PubMed]

Tabarias, H.

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).
[CrossRef] [PubMed]

Tanimoto, N.

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).
[CrossRef]

Taylor, P. M.

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).
[CrossRef] [PubMed]

Tazelaar, J.

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).
[CrossRef] [PubMed]

Teusner, J. T.

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).
[CrossRef]

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).
[CrossRef]

Thrasher, A. J.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Timmers, A. M.

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).
[CrossRef]

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).
[CrossRef]

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.

Tonagel, F.

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).
[CrossRef]

Vaquero, M. I.

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).
[CrossRef] [PubMed]

Wang, Y.

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).
[CrossRef] [PubMed]

Weber, B. H.

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).
[CrossRef]

Weber, M.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

Wilson, J. M.

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).
[CrossRef] [PubMed]

Windsor, E. A.

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).
[CrossRef]

Xiao, W.

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).
[CrossRef]

Xiao, X.

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).
[CrossRef]

Yamada, E. S.

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).
[CrossRef]

Yan, Z.

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).
[CrossRef] [PubMed]

Yang, G. S.

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).
[CrossRef] [PubMed]

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).
[CrossRef] [PubMed]

Yann, P.

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

Zabner, J.

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).
[CrossRef] [PubMed]

Zack, D. J.

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).
[CrossRef] [PubMed]

Zaknich, T.

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).
[CrossRef] [PubMed]

Zeiss, C. J.

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).
[CrossRef]

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).
[CrossRef] [PubMed]

Zeng, Y.

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).
[CrossRef] [PubMed]

Zhang, D.

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).
[CrossRef] [PubMed]

Zhang, Q.

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).
[CrossRef] [PubMed]

Zolotukhin, S.

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).
[CrossRef] [PubMed]

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).
[PubMed]

Arch. Ophthalmol. (Chicago) (1)

G. LeMeur, M. Weber, P. Yann, A. Mendes-Madeira, D. Nivard, J. Deschamps, P. 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).

Br. Behavior Evol. (1)

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).
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Gene Ther. (1)

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Figures (3)

Fig. 1
Fig. 1

Molecular map of rAAV.CHOPS2053.GFP. TR = terminal repeats; LCR = locus control region from upstream of the human L opsin gene; PP = proximal promoter of the human L opsin gene; SD SA = mini -intron with splice donor (SD) and acceptor sites (SA) required for efficient transcription and translation in eukaryotic cells; GFP = cDNA encoding green fluorescent protein; PA 1 = an SV40 polyadenylation signal for efficient translation in eukaryotic cells.

Fig. 2
Fig. 2

rAAV.CHOPS2053.GFP transduces cone photoreceptors in the squirrel monkey. Near the injection site, which was placed just inferior to the fovea, GFP fluorescence was observed within cones. At the near peripheral location of the field shown, the cone density is high and there are relatively few rods. At this eccentricity from the fovea in the primate retina, rods and cones are easily distinguishable because of marked anatomical differences, with cone inner segments being 2 to 3 times wider in diameter than rods. At a more peripheral location distal to the injection site (inset), cones are even more distinct with their larger diameters, and fewer cells were transduced as evidenced by isolated cones expressing GFP surrounded by nontransduced cells. In the near periphery, it appears that a majority of cones express GFP. Within the same field, there is also an occasional cell with a narrow profile which expresses GFP. This may be spurious expression of GFP in rods. In locations distal from the injection site where occasional GFP expressing cones are observed, no suspect rods expressing GFP were seen. Abbreviations: OS = outer segment; IS = inner segment; ONL = outer nuclear layer.

Fig. 3
Fig. 3

GFP fluorescence imaged in a living monkey using the RetCam II. All images show the right eye of animal 265750. Images in panels (A)–(D) were obtained using a halogen bulb; images in panels (E)–(H) were obtained using a xenon bulb. In panels (D)–(H), images were obtained with a green filter ( 510 nm cutoff) placed between the light source and the camera for detecting GFP fluorescence. (A) Fundus image taken immediately before the injection procedure. Blood vessels provide landmarks that allow comparisons to be made across images. (B) Same retinal area as panel (A), which corresponds to the location of the injection that was made inferior and temporal to the optic nerve head, imaged directly following the injection procedure. A large bleb of virus-containing solution that was made underneath the retina is visible. The bleb and retinal landmarks are labeled in (C). (D) Image taken 9   weeks postinjection using a 130° lens. (E) Same retinal area as panel D showing a small, roughly J-shaped area of GFP expression. (F) Image taken 12   weeks postinjection using a high-magnification 30° lens. The smaller lens was found to prevent unfiltered light at the edges, thus eliminating the greenish background seen in the image shown in panel (E). In images (F)–(H), all of the green light is coming strictly from the GFP fluorescence. (G) Same retinal area shown in panel (F), imaged at 20   weeks postinjection. (H) An image taken at 24   weeks postinjection. (I) Graph of the relative intensity of the GFP fluorescence in images (E)–(H). Pixel intensities are plotted on a scale in which the lowest possible intensity equals zero and the highest equals 100.

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