Dr. Lucie Guo from Stanford University awarded $70,000 Knights Templar Eye Foundation Grant for a Novel Gene Therapy for Pediatric Optic Nerve Disorders

The optic nerve is a critical structure for vision; it transmits signals from the lightsensing neural layer of the eye to the brain. The optic nerve is formed by axons of retinal ganglion cells, which are neurons that are highly susceptible to damage in several types of incurable blinding diseases in children. There is an urgent need to develop sight-restoring therapeutics for these optic nerve diseases. Gene therapy is already in clinical use for pediatric eye disease, but using gene therapy for optic nerve disorders is currently hampered by multiple technical barriers.

Dr. Guo proposes to use a new CRISPR-based gene regulation technology to overcome these barriers and develop a novel type of gene therapy, with the longterm goal of generating new treatments for blinding diseases in children.