Optogenetic Therapy

Patients with blinding disorders due to retinal defects often have no or limited treatment options. Loss of sight dramatically distorts an individual’s interaction with the physical and social environment, leading to significant co-morbidity, reduced mobility and mental isolation. The affected population is large with an estimated 3 million Americans completely blind due to retinal disorders. The burden on the health care system is substantial, as is the societal burden. We aim to develop optogenetic gene therapy as a treatment to restore vision in blind eyes. Vision would be brought to totally blind retinas by rendering the cells remaining in the retina (once the photoreceptors have deteriorated) light-sensitive. Optogenetic proteins have been identified in ancient bacteria and algae. These are light-gated ionic channels or pumps, which can be used to activate or inhibit neuronal activity upon light stimulation. They can be readily packaged in recombinant AAV particles. Proof-of-concept that light sensitivity can be restored to the retina using optogenetic therapy has been demonstrated using rodent models. We aim to optimize the various components of optogenetic therapy so that it can be tested in large animals and ultimately in humans who are completely blind. We are carrying out optogenetic gene therapy studies as an international collaboration with individuals in Europe (Drs. Sahel, Roska, Picaud, Cronin, Bamberg) and in the USA (Drs. Vandenberghe, Boyden)