Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina
- 11 April 2007
- journal article
- Published by Optica Publishing Group in Journal of the Optical Society of America A
- Vol. 24 (5) , 1411-1416
- https://doi.org/10.1364/josaa.24.001411
Abstract
Recombinant adeno-associated virus (rAAV) is a promising vector for gene therapy of photoreceptor-based diseases. Previous studies have demonstrated that rAAV serotypes 2 and 5 can transduce both rod and cone photoreceptors in rodents and dogs, and it can target rods, but not cones in primates. Here we report that using a human cone-specific enhancer and promoter to regulate expression of a green fluorescent protein (GFP) reporter gene in an rAAV-5 vector successfully targeted expression of the reporter gene to primate cones, and the time course of GFP expression was able to be monitored in a living animal using the RetCam II digital imaging system.Keywords
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