Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina. J Opt Soc Am A Opt Image Sci Vis 2007 May;24(5):1411-6
Date
04/13/2007Pubmed ID
17429487DOI
10.1364/josaa.24.001411Scopus ID
2-s2.0-34249721535 (requires institutional sign-in at Scopus site) 27 CitationsAbstract
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.
Author List
Mancuso K, Hendrickson AE, Connor TB Jr, Mauck MC, Kinsella JJ, Hauswirth WW, Neitz J, Neitz MAuthor
Thomas B. Connor MD Professor in the Ophthalmology and Visual Sciences department at Medical College of WisconsinMESH terms used to index this publication - Major topics in bold
AdenoviridaeAnimals
Gene Expression
Gene Targeting
Gene Transfer Techniques
Green Fluorescent Proteins
Recombinant Proteins
Retina
Retinal Cone Photoreceptor Cells
Sciuridae
