caa a22 4500 397575092 CHVBK 20180308164853.0 cr unu---uuuuu 161202e199605 xx s 000 0 eng 10.1093/hmg/5.5.591 doi (NATIONALLICENCE)oxford-10.1093/hmg/5.5.591 Gene Transfer into the Mouse Retina Mediated by an Adeno-Associated Viral Vector [Elektronische Daten] [Robin R. Ali, Martin B. Reichel, Adrian J. Thrasher, Roland J. Levinsky, Christine Kinnon, Naheed Kanuga, David M. Hunt, Shomi S. Bhattacharya] Gene transfer to photoreceptor cells may provide a means for arresting the retinal degeneration that is characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). However, transduction of photoreceptors has to date been inefficient, and further limited by toxicity and immune responses directed against vector-specific proteins. An alternative vector system based on adeno-associated virus (AAV) may obviate these problems, and may be useful for transduction of neuronal cells. In this study we have demonstrated successful transduction of all layers of the neuroretina as well as the retinal pigment epithelium (RPE) following subretinal injection of recombinant AAV particles encoding lac Z. Furthermore, the efficiency of transduction of photoreceptors is significantly higher than that achieved with an equivalent adenoviral vector. This is the first report showing that AAV is capable of transducing photoreceptor cells and supports the use of this vector system for gene therapy of retinal diseases such as RP. © 1996 Oxford University Press Ali Robin R. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut Reichel Martin B. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut Thrasher Adrian J. Division of Cell and Molecular Biology, Institute of Child Health, University College London, Guilford St., London WC1N 1EH, UK aut Levinsky Roland J. Division of Cell and Molecular Biology, Institute of Child Health, University College London, Guilford St., London WC1N 1EH, UK aut Kinnon Christine Division of Cell and Molecular Biology, Institute of Child Health, University College London, Guilford St., London WC1N 1EH, UK aut Kanuga Naheed Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut Hunt David M. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut Bhattacharya Shomi S. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut Human Molecular Genetics Oxford University Press 5/5(1996-05), 591-594 0964-6906 5:5<591 1996 5 hmg https://doi.org/10.1093/hmg/5.5.591 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1093/hmg/5.5.591 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Ali Robin R. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut NATIONALLICENCE 700 1- Reichel Martin B. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut NATIONALLICENCE 700 1- Thrasher Adrian J. Division of Cell and Molecular Biology, Institute of Child Health, University College London, Guilford St., London WC1N 1EH, UK aut NATIONALLICENCE 700 1- Levinsky Roland J. Division of Cell and Molecular Biology, Institute of Child Health, University College London, Guilford St., London WC1N 1EH, UK aut NATIONALLICENCE 700 1- Kinnon Christine Division of Cell and Molecular Biology, Institute of Child Health, University College London, Guilford St., London WC1N 1EH, UK aut NATIONALLICENCE 700 1- Kanuga Naheed Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut NATIONALLICENCE 700 1- Hunt David M. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut NATIONALLICENCE 700 1- Bhattacharya Shomi S. Department of Molecular Genetics, Institute of Ophthalmology, University College London, Bath St., London EC1V 9EL, UK aut NATIONALLICENCE 773 0- Human Molecular Genetics Oxford University Press 5/5(1996-05), 591-594 0964-6906 5:5<591 1996 5 hmg Metadata rights reserved CC BY-NC-4.0 http://creativecommons.org/licenses/by-nc/4.0 nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-oxford