Researchers based at INSERM, Sorbonne UniversitÃƒ©s 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.
Intravitreal AAV delivery of optogenetic construct to ON bipolar cells rescues visual function
- by swdadmin