The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.

ABSTRACT: Adeno-associated virus (AAV) has proven an effective gene delivery vehicle for the treatment of retinal disease. Ongoing clinical trials using a serotype 2 AAV vector to express RPE65 in the retinal pigment epithelium have proven safe and effective. While many proof-of-concept studies in animal models of retinal disease have suggested that gene transfer to the neural retina will also be effective, a photoreceptor-targeting AAV vector has yet to be used in the clinic, principally because a vector that efficiently but exclusively targets all primate photoreceptors has yet to be demonstrated. Here, we evaluate a serotype 5 AAV vector containing the human rhodopsin kinase (hGRK1) promoter for its ability to target transgene expression to rod and cone photoreceptors when delivered subretinally

SUBMITTER: Boye SE 

PROVIDER: S-EPMC3472519 | biostudies-literature | 2012 Oct

REPOSITORIES: biostudies-literature