In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous
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- 12 June 2013
- journal article
- research article
- Published by American Association for the Advancement of Science (AAAS) in Science Translational Medicine
- Vol. 5 (189), 189ra76
- https://doi.org/10.1126/scitranslmed.3005708
Abstract
Injection of a new gene therapy vector into the easily accessible vitreous transduced the entire retina and rescued disease phenotypes.Keywords
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