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Research Article

Evaluation of recombinant adeno-associated virus as a gene transfer vector for the retina

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Pages 949-956 | Published online: 02 Jul 2009

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Read on this site (4)

Sheng-Hai Zhang*Ji-Hong Wu*Xiao-Bing Wu*Xiao-Yan Dong, Xin-Jian Liu & Chuan-Yuan Li. (2008) Distinctive Gene Transduction Efficiencies of Commonly Used Viral Vectors in the Retina. Current Eye Research 33:1, pages 81-90.
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Wei-Chi Wu, Chi-Chun Lai, Show-Li Chen, Ming-Hui Sun, Xiao Xiao, Tun-Lu Chen, Ken-Kuo Lin, Shu-Wen Kuo & Yeou-Ping Tsao. (2005) Long-Term Safety of GDNF Gene Delivery in the Retina. Current Eye Research 30:8, pages 715-722.
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Eliisa Mannermaa, Seppo Rönkkö, Marika Ruponen, Mika Reinisalo & Arto Urtti. (2005) Long-Lasting Secretion of Transgene Product from Differentiated and Filter-Grown Retinal Pigment Epithelial Cells After Nonviral Gene Transfer. Current Eye Research 30:5, pages 345-353.
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Trevor J McFarland, Yi Zhang, Binoy Appukuttan & J Timothy Stout. (2004) Gene therapy for proliferative ocular diseases. Expert Opinion on Biological Therapy 4:7, pages 1053-1058.
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Articles from other publishers (51)

Rachel Atkinson, Jacqueline Leung, James Bender, Matthew Kirkcaldie, James Vickers & Anna King. (2021) TDP-43 mislocalization drives neurofilament changes in a novel model of TDP-43 proteinopathy. Disease Models & Mechanisms 14:2.
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Marianthi Karali, Irene Guadagnino, Elena Marrocco, Rossella De Cegli, Annamaria Carissimo, Mariateresa Pizzo, Simona Casarosa, Ivan Conte, Enrico Maria Surace & Sandro Banfi. (2020) AAV-miR-204 Protects from Retinal Degeneration by Attenuation of Microglia Activation and Photoreceptor Cell Death. Molecular Therapy - Nucleic Acids 19, pages 144-156.
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Elena Herrera-CarrilloYing Poi LiuBen Berkhout. (2017) Improving miRNA Delivery by Optimizing miRNA Expression Cassettes in Diverse Virus Vectors. Human Gene Therapy Methods 28:4, pages 177-190.
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Preethi SomasundaramGlenn R. WyrickDiego Carlos FernandezAlireza GhahariCindy M. PinhalMelissa Simmonds RichardsonAlan C. RuppLihong CuiZhijian WuR. Lane BrownTudor Constantin BadeaSamer Hattar & Phyllis R. Robinson. (2017) C-terminal phosphorylation regulates the kinetics of a subset of melanopsin-mediated behaviors in mice. Proceedings of the National Academy of Sciences 114:10, pages 2741-2746.
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Sandy S.C. Hung, Tristan McCaughey, Olivia Swann, Alice Pébay & Alex W. Hewitt. (2016) Genome engineering in ophthalmology: Application of CRISPR/Cas to the treatment of eye disease. Progress in Retinal and Eye Research 53, pages 1-20.
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Michelle E. McClements & Robert E. MacLaren. 2015. Translating Gene Therapy to the Clinic. Translating Gene Therapy to the Clinic 173 189 .
Michelle E. McClements & Robert E. MacLaren. (2013) Gene therapy for retinal disease. Translational Research 161:4, pages 241-254.
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Ying Poi Liu & Ben Berkhout. (2011) miRNA cassettes in viral vectors: Problems and solutions. Biochimica et Biophysica Acta (BBA) - Gene Regulatory Mechanisms 1809:11-12, pages 732-745.
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C Mussolino, M della Corte, S Rossi, F Viola, U Di Vicino, E Marrocco, S Neglia, M Doria, F Testa, R Giovannoni, M Crasta, M Giunti, E Villani, M Lavitrano, M L Bacci, R Ratiglia, F Simonelli, A Auricchio & E M Surace. (2011) AAV-mediated photoreceptor transduction of the pig cone-enriched retina. Gene Therapy 18:7, pages 637-645.
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Lauren E Mays & James M Wilson. (2011) The Complex and Evolving Story of T cell Activation to AAV Vector-encoded Transgene Products. Molecular Therapy 19:1, pages 16-27.
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Kati Kinnunen, Giedrius Kalesnykas, Anssi J. Mähönen, Svetlana Laidinen, Liisa Holma, Tommi Heikura, Kari Airenne, Hannu Uusitalo & Seppo Ylä‐Herttuala. (2009) Baculovirus is an efficient vector for the transduction of the eye: comparison of baculovirus‐ and adenovirus‐mediated intravitreal vascular endothelial growth factor D gene transfer in the rabbit eye. The Journal of Gene Medicine 11:5, pages 382-389.
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M Natkunarajah, P Trittibach, J McIntosh, Y Duran, S E Barker, A J Smith, A C Nathwani & R R Ali. (2007) Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8. Gene Therapy 15:6, pages 463-467.
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Inchan Kwon & David V. Schaffer. (2007) Designer Gene Delivery Vectors: Molecular Engineering and Evolution of Adeno-Associated Viral Vectors for Enhanced Gene Transfer. Pharmaceutical Research 25:3.
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Takao Hashimoto. 2008. Recent Advances in Retinal Degeneration. Recent Advances in Retinal Degeneration 113 119 .
Mariacarmela Allocca, Claudio Mussolino, Maria Garcia-Hoyos, Daniela Sanges, Carolina Iodice, Marco Petrillo, Luk H. Vandenberghe, James M. Wilson, Valeria Marigo, Enrico M. Surace & Alberto Auricchio. (2007) Novel Adeno-Associated Virus Serotypes Efficiently Transduce Murine Photoreceptors. Journal of Virology 81:20, pages 11372-11380.
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Astra DinculescuLyudmyla GlushakovaSeok-Hong MinWilliam W. Hauswirth. (2005) Adeno-Associated Virus-Vectored Gene Therapy for Retinal Disease. Human Gene Therapy 16:6, pages 649-663.
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LINGYUN CHENG, MITSUKO TOYOGUCHI, DAVID J. LOONEY, JEFFERY LEE, MARIE C. DAVIDSON & WILLIAM R. FREEMAN. (2005) EFFICIENT GENE TRANSFER TO RETINAL PIGMENT EPITHELIUM CELLS WITH LONG-TERM EXPRESSION. Retina 25:2, pages 193-201.
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K R G Martin & H A Quigley. (2004) Gene therapy for optic nerve disease. Eye 18:11, pages 1049-1055.
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Anne K. Hennig, Judith Mosinger Ogilvie, Kevin K. Ohlemiller, Adrian M. Timmers, William W. Hauswirth & Mark S. Sands. (2004) AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice. Molecular Therapy 10:1, pages 106-116.
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Stefan Isenmann, Christian Schmeer & Alexandra Kretz. (2004) How to keep injured CNS neurons viable—strategies for neuroprotection and gene transfer to retinal ganglion cells. Molecular and Cellular Neuroscience 26:1, pages 1-16.
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Jie Zhang, Qing Shan, Ping Ma, Yanming Jiang, Peng Chen, Jingxia Wen, You Zhou, Huanwen Qian & Xuetao Pei. (2004) Differentiation potential of bone marrow mesenchymal stem cells into retina in normal and laser-injured rat eye. Science in China Series C: Life Sciences 47:3, pages 241-250.
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Wei-Yong Shen, Yvonne K.Y. Lai, Chooi-May Lai & P.Elizabeth Rakoczy. (2004) Impurity of recombinant adeno-associated virus type 2 affects the transduction characteristics following subretinal injection in the rat. Vision Research 44:4, pages 339-348.
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Andrew J. Lotery, Grace S. Yang, Robert F. Mullins, Stephen R. Russell, Michael Schmidt, Edwin M. Stone, Jonathan D. Lindbloom, John A. Chiorini, Robert M. Kotin & Beverly L. Davidson. (2003) Adeno-Associated Virus Type 5: Transduction Efficiency and Cell-Type Specificity in the Primate Retina. Human Gene Therapy 14:17, pages 1663-1671.
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Wei‐Yong Shen, Chooi‐May Lai, Yvonne K. Y. Lai, Dan Zhang, Tammy Zaknich, Erika N. Sutanto, Ian J. Constable & P. Elizabeth Rakoczy. (2003) Practical considerations of recombinant adeno‐associated virus‐mediated gene transfer for treatment of retinal degenerations. The Journal of Gene Medicine 5:7, pages 576-587.
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Alberto Auricchio. (2003) Pseudotyped AAV vectors for constitutive and regulated gene expression in the eye. Vision Research 43:8, pages 913-918.
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Joyce Tombran-Tink. 2003. Ophthalmic Drug Delivery Systems, Second Edition. Ophthalmic Drug Delivery Systems, Second Edition 535 608 .
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P. Elizabeth Rakoczy, Chooi-May Lai, Meaghan J. T. Yu, Dru M. Daniels, Meliha Brankov, Ben C. Rae, Chris W. Stoddart, Nigel L. Barnett, Matthew T. Martin-Iverson, T. Michael Redmond, Kristina Narfstrom, Xiaohuai Zhou & Ian J. Constable. 2003. Retinal Degenerations. Retinal Degenerations 431 438 .
Thucanh T. Ho, Albert M. Maguire, Gustavo D. Aguirre, Enrico M. Surace, Vibha Anand, Yong Zeng, Anna Salvetti, John J. Hopwood, Mark E. Haskins & Jean Bennett. (2002) Phenotypic rescue after adeno‐associated virus‐mediated delivery of 4‐sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI. The Journal of Gene Medicine 4:6, pages 613-621.
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Keith R.G Martin, Ronald L Klein & Harry A Quigley. (2002) Gene delivery to the eye using adeno-associated viral vectors. Methods 28:2, pages 267-275.
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Edward Chaum & Mark P Hatton. (2002) Gene Therapy for Genetic and Acquired Retinal Diseases. Survey of Ophthalmology 47:5, pages 449-469.
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A.R. Harvey, W. Kamphuis, R. Eggers, N.A. Symons, B. Blits, S. Niclou, G.J. Boer & J. Verhaagen. (2002) Intravitreal Injection of Adeno-associated Viral Vectors Results in the Transduction of Different Types of Retinal Neurons in Neonatal and Adult Rats: A Comparison with Lentiviral Vectors. Molecular and Cellular Neuroscience 21:1, pages 141-157.
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Todd A. Derksen, Sybille L. Sauter & Beverly L. Davidson. (2002) Feline immunodeficiency virus vectors. Gene transfer to mouse retina following intravitreal injection. The Journal of Gene Medicine 4:5, pages 463-469.
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Grace S. Yang, Michael Schmidt, Ziying Yan, Jonathan D. Lindbloom, Thomas C. Harding, Brian A. Donahue, John F. Engelhardt, Robert Kotin & Beverly L. Davidson. (2002) Virus-Mediated Transduction of Murine Retina with Adeno-Associated Virus: Effects of Viral Capsid and Genome Size. Journal of Virology 76:15, pages 7651-7660.
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Alberto Auricchio, Victor M Rivera, Tim Clackson, Erin E O'Connor, Albert M Maguire, Michael J Tolentino, Jean Bennett & James M Wilson. (2002) Pharmacological Regulation of Protein Expression from Adeno-Associated Viral Vectors in the Eye. Molecular Therapy 6:2, pages 238-242.
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Li Cheng, Przemyslaw Sapieha, Pavla Kittlerová, William W. Hauswirth & Adriana Di Polo. (2002) TrkB Gene Transfer Protects Retinal Ganglion Cells from Axotomy-Induced Death In Vivo . The Journal of Neuroscience 22:10, pages 3977-3986.
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