Glaucoma is a progressive optic neuropathy and a major cause of visual impairment
worldwide. Neuroprotective therapies for glaucoma aim to ameliorate retinal ganglion cell …

Vectors based on adeno-associated virus serotype 2 (AAV2) have been used extensively in
many gene-delivery applications, including several successful clinical trials for one type of …

Previous work established retinal expression of channelrhodopsin-2 (ChR2), an algal cation
channel gated by light, restored physiological and behavioral visual responses in otherwise …

Purpose.: Adeno-associated virus serotype 2 (AAV2) has been shown to be effective in
transducing inner retinal neurons after intravitreal injection in several species. However …

The retinal degeneration 10 (rd10) mouse is a well-characterized model of autosomal
recessive retinitis pigmentosa (RP), which carries a spontaneous mutation in the β subunit of …

Mutations in over 80 identified genes can induce apoptosis in photoreceptors, resulting in
blindness with a prevalence of 1 in 3,000 individuals. This broad genetic heterogeneity of …

Therapies currently used for hemophilia involve injection of protein concentrates that are
expensive, invasive and associated with side effects such as development of neutralizing …

Patients with Bardet-Biedl syndrome (BBS) experience severe retinal degeneration as a
result of impaired photoreceptor transport processes that are not yet fully understood. To …

Purpose.: The authors previously showed that subretinal delivery of AAV5 vectors containing
murine guanylate cyclase-1 (GC1) cDNA driven by either photoreceptor-specific (hGRK1) or …

Purpose With the increasing number of retinal gene-based therapies and therapeutic
constructs, in vitro bioassays characterizing vector transduction efficiency and quality are …