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
Database: MEDLINE Ultimate
Description
ISSN:1476-5462
DOI:10.1038/s41434-023-00436-8