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Adeno‐associated virus 8‐mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models |
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| Authors: | Aaron Black Vidyullatha Vasireddy Daniel C. Chung Albert M. Maguire Rajashekhar Gaddameedi Tania Tolmachova Miguel Seabra Jean Bennett |
| Affiliation: | 1. F.M. Kirby Center for Molecular Ophthalmology and Center for Advanced Retinal and Ophthalmic Therapeutics, Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia, PA, USA;2. Molecular Medicine, Imperial College London, London, UK |
| Abstract: | |
| Keywords: | adeno‐associated virus animal models choroideremia gene therapy in vitro models retina |