Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina |
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Authors: | Mancuso Katherine Hendrickson Anita E Connor Thomas B Mauck Matthew C Kinsella James J Hauswirth William W Neitz Jay Neitz Maureen |
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Affiliation: | Department of Cell Biology, Neurobiology & Anatomy, Medical College of Wisconsin, Milwaukee 53226, USA. |
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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. |
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