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While Adeno-Associated Viruses (AAVs) shows great promise in gene therapy applications for the treatment of inherited and acquired retinal diseases, a significant challenge to date has been the limitation of AAV vectors to transduce non-permissive retinal cells and to express in a single cell type.
The inventors have generated novel AAVs that have enhanced specificity for retinal bipolar cells, which are involved in processing visual signals and have become the focus of attention for optogenetic therapies for the retina, such as retinitis pigmentosa. The new viruses could be used to develop treatments for glaucoma and neovascular conditions adversely affecting vision (choroidal neovascularization, diabetic retinopathy, retinopathy of prematurity, sickle cell retinopathy, retinal vein occlusion).
Pending worldwide rights
Docket # Z6471