Multi-Characteristic Opsin enabled Vision Restoration

摘要

Photodegenerative retinal diseases such as retinitis pigmentosa (RP) and dry age related macular degeneration (dry-AMD) lead to loss of vision in millions of individuals. Currently, no surgical or medical treatment is available though optogenetic therapies are in clinical development. Here, we demonstrate vision restoration using Multi-Characteristics Opsin (MCO1) in animal models with photo-degenerated retina. MCO1 is reliably delivered to specific retinal cells via intravitreal injection of Adeno-Associated Virus, leading to significant improvement in visually guided behavior conducted using a radial-arm water maze. The time to reach platform significantly reduced after delivery of MCO1. Notably, the improvement in visually guided behavior was observed even at light intensity levels orders of magnitude lower than that required for Channelrhodopsin-2 opsin. Chronic light exposure study showed that chronic light exposure did not compromise viability of vMCO1-treated retina. Safe virus-mediated MCO1-delivery has potential for effective gene therapy of diverse retinal degenerations in patients.