Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina.
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.
Mancuso
Katherine
K
Department of Cell Biology, Neurobiology & Anatomy, Medical College of Wisconsin, Milwaukee 53226, USA.
Hendrickson
Anita E
AE
Connor
Thomas B
TB
Mauck
Matthew C
MC
Kinsella
James J
JJ
Hauswirth
William W
WW
Neitz
Jay
J
Neitz
Maureen
M
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
D011994:Recombinant Proteins; D049452:Green Fluorescent Proteins