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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes.

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Title: AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes.
Authors: Chaqour B; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA., Duong TT; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.; GlaxoSmithKline, Collegeville, PA, 19426, USA., Yue J; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA., Liu T; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.; Spark Therapeutics, Philadelphia, PA, 19104, USA., Camacho D; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA., Dine KE; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA., Esteve-Rudd J; Gyroscope Therapeutics Limited, a Novartis Company, London, N7 9AS, UK., Ellis S; Gyroscope Therapeutics Limited, a Novartis Company, London, N7 9AS, UK., Bennett J; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA., Shindler KS; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA. kenneth.shindler@pennmedicine.upenn.edu.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA. kenneth.shindler@pennmedicine.upenn.edu.; Department of Neurology, University of Pennsylvania, Philadelphia, PA, 19104, USA. kenneth.shindler@pennmedicine.upenn.edu., Ross AG; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA. ahmara.ross@pennmedicine.upenn.edu.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA. ahmara.ross@pennmedicine.upenn.edu.; Department of Neurology, University of Pennsylvania, Philadelphia, PA, 19104, USA. ahmara.ross@pennmedicine.upenn.edu.
Source: Gene therapy [Gene Ther] 2024 Mar; Vol. 31 (3-4), pp. 175-186. Date of Electronic Publication: 2024 Jan 10.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE
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  Data: <searchLink fieldCode="AU" term="%22Chaqour+B%22">Chaqour B</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Duong+TT%22">Duong TT</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.; GlaxoSmithKline, Collegeville, PA, 19426, USA.<br /><searchLink fieldCode="AU" term="%22Yue+J%22">Yue J</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Liu+T%22">Liu T</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.; Spark Therapeutics, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Camacho+D%22">Camacho D</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Dine+KE%22">Dine KE</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Esteve-Rudd+J%22">Esteve-Rudd J</searchLink>; Gyroscope Therapeutics Limited, a Novartis Company, London, N7 9AS, UK.<br /><searchLink fieldCode="AU" term="%22Ellis+S%22">Ellis S</searchLink>; Gyroscope Therapeutics Limited, a Novartis Company, London, N7 9AS, UK.<br /><searchLink fieldCode="AU" term="%22Bennett+J%22">Bennett J</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA.<br /><searchLink fieldCode="AU" term="%22Shindler+KS%22">Shindler KS</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA. kenneth.shindler@pennmedicine.upenn.edu.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA. kenneth.shindler@pennmedicine.upenn.edu.; Department of Neurology, University of Pennsylvania, Philadelphia, PA, 19104, USA. kenneth.shindler@pennmedicine.upenn.edu.<br /><searchLink fieldCode="AU" term="%22Ross+AG%22">Ross AG</searchLink>; Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA, 19104, USA. ahmara.ross@pennmedicine.upenn.edu.; F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, 19104, USA. ahmara.ross@pennmedicine.upenn.edu.; Department of Neurology, University of Pennsylvania, Philadelphia, PA, 19104, USA. ahmara.ross@pennmedicine.upenn.edu.
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  Data: <searchLink fieldCode="JN" term="%229421525%22">Gene therapy</searchLink> [Gene Ther] 2024 Mar; Vol. 31 (3-4), pp. 175-186. <i>Date of Electronic Publication: </i>2024 Jan 10.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Nature+Publishing+Group%22">Nature Publishing Group </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>9421525 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1476-5462 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209697128%22">09697128 </searchLink><i>NLM ISO Abbreviation: </i>Gene Ther <i>Subsets: </i>MEDLINE
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