Intravitreal AAV delivery of optogenetic construct to ON bipolar cells rescues visual function

Researchers based at INSERM, Sorbonne Universités and CNRS in Paris have demonstrated optogenetic rescue of visual function in models of retinal degeneration using intra-vitreally delivered AAV. The studies used a modified channelrhodopsin protein designed for expression in ON bipolar cells located within deep layers of the retina. The proof-of-concept results indicate that optogenetic reactivation of ON-bipolar cells may be a viable option for restoring vision in patients with late-stage disease. In addition, the use of engineered AAV delivery vehicles that can be injected intra-vitreally, rather than sub-retinally, may significantly change the costs associated with the promising ocular gene therapy market.