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Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina
Katherine Mancuso, Anita E. Hendrickson, Thomas B. Connor, Jr., Matthew C. Mauck, James J. Kinsella, William W. Hauswirth, Jay Neitz, and Maureen Neitz »View Author Affiliations
Katherine Mancuso,1
Anita E. Hendrickson,2
Thomas B. Connor,3
Matthew C. Mauck,4
James J. Kinsella,4
William W. Hauswirth,5
Jay Neitz,6
and Maureen Neitz6
1Departments of Cell Biology, Neurobiology & Anatomy, and Ophthalmology, Medical College of Wisconsin, 925 North 87th Street, Milwaukee, Wisconsin 53226, USA
2Department of Biological Structure, University of Washington, Box 357420, Seattle, Washington 98195, USA
3Department of Ophthalmology, Medical College of Wisconsin, 925 North 87th Street, Milwaukee, Wisconsin 53226, USA
4Departments of Cell Biology, Neurobiology & Anatomy, and Ophthalmology, Medical College of Wisconsin, 925 North 87th Street, Milwaukee, Wisconsin 53226, USA
5Department of Ophthalmology and Powell Gene Therapy Center, University of Florida, Gainesville, Florida 32610, USA
6Department of Ophthalmology, Medical College of Wisconsin, 925 North 87th Street, Milwaukee, Wisconsin 53226, USA
JOSA A, Vol. 24, Issue 5, pp. 1411-1416 (2007)
http://dx.doi.org/10.1364/JOSAA.24.001411
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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
OCIS Codes
(110.0110) Imaging systems : Imaging systems
(180.0180) Microscopy : Microscopy
(180.2520) Microscopy : Fluorescence microscopy
(330.0330) Vision, color, and visual optics : Vision, color, and visual optics
(330.1720) Vision, color, and visual optics : Color vision
(330.5310) Vision, color, and visual optics : Vision - photoreceptors
ToC Category:
Clinical Applications of Retinal Imaging
History
Original Manuscript: September 20, 2006
Revised Manuscript: November 21, 2006
Manuscript Accepted: November 21, 2006
Published: April 11, 2007
Virtual Issues
Vol. 2, Iss. 6 Virtual Journal for Biomedical Optics
Citation
Katherine Mancuso, Anita E. Hendrickson, Thomas B. Connor, Jr., Matthew C. Mauck, James J. Kinsella, William W. Hauswirth, Jay Neitz, and Maureen Neitz, "Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina," J. Opt. Soc. Am. A 24, 1411-1416 (2007)
http://www.opticsinfobase.org/josaa/abstract.cfm?URI=josaa-24-5-1411
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- 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]
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- 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]
- 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]
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- 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]
- 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]
- 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]
- 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]
- 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]
- 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]
- 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]
- 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]
- 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]
- 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. 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]
- 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]
- 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]
- 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]
- 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.
- 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. 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]
- 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]
- 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).
- 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]
- 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. 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. 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]
- 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]
- 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]
- 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]
- 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).
- 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]
- 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]
- 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]
- 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. 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]
- 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]
- 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]
Arch. Ophthalmol. (Chicago)
- 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.
- 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]
Gene Ther.
- 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]
Graefe's Arch. Clin. Exp. Ophthalmol.
- 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).
Hum. Gene Ther.
- 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]
- 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]
- 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]
Invest. Ophthalmol. Visual Sci.
- 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. Comp. Neurol.
- 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]
J. Gene Med.
- 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]
J. Opt. Soc. Am.
- 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. Virol.
- 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. 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. 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]
Methods Enzymol.
- 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]
Mol. Therapy
- 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]
- 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]
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Nat. Genet.
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