title: Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina.
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.
authors: Department of Cell Biology, Neurobiology & Anatomy, Medical College of Wisconsin, Milwaukee 53226, USA.
authors: Katherine
authors: K
authors: Mancuso
authors: Anita E
authors: AE
authors: Hendrickson
authors: Thomas B
authors: TB
authors: Connor
authors: Matthew C
authors: MC
authors: Mauck
authors: James J
authors: JJ
authors: Kinsella
authors: William W
authors: WW
authors: Hauswirth
authors: Jay
authors: J
authors: Neitz
authors: Maureen
authors: M
authors: Neitz
mesh_terms: D000256:Adenoviridae / Q000235:genetics*; D000818:Animals; D015870:Gene Expression / Q000502:physiology*; D018390:Gene Targeting / Q000379:methods*; D018014:Gene Transfer Techniques; D049452:Green Fluorescent Proteins / Q000235:genetics* / Q000378:metabolism*; D011994:Recombinant Proteins / Q000378:metabolism; D012160:Retina / Q000378:metabolism; D017949:Retinal Cone Photoreceptor Cells / Q000378:metabolism*; D012589:Sciuridae
chemical_list: D011994:Recombinant Proteins; D049452:Green Fluorescent Proteins