Ontology highlight
ABSTRACT:
SUBMITTER: Boye SE
PROVIDER: S-EPMC3472519 | biostudies-literature | 2012 Oct
REPOSITORIES: biostudies-literature
Boye Shannon E SE Alexander John J JJ Boye Sanford L SL Witherspoon Clark D CD Sandefer Kristen J KJ Conlon Thomas J TJ Erger Kirsten K Sun Jingfen J Ryals Renee R Chiodo Vince A VA Clark Mark E ME Girkin Christopher A CA Girkin Christopher A CA Hauswirth William W WW Gamlin Paul D PD
Human gene therapy 20120920 10
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 ...[more]