Studying Gene Regulation Networks in Retinal Ganglion Cells for Novel Neuroprotective Targets

Glaucoma is the most common cause of irreversible blindness and by 2040 will affect more than 100 million people between 40 and 80 years of age, worldwide. Glaucoma is characterized by optic nerve neuropathy with retinal ganglion cell (RGC) axon degeneration followed by progressive RGC death. Understanding gene regulation mechanisms that are associated with RGCs at normal function, under disease, or after treatment, is essential for identifying novel therapeutic targets and innovative and efficient neural repair strategies. We are taking advantage of newly developed genetic tools to elucidate the comprehensive gene regulatory networks that will serve as a blueprint for developing novel and effective neuroprotectants for glaucoma.

Our long-term goal is to develop safe and effective neuroprotective agents for glaucoma patients, through our efforts to elucidate the molecular mechanisms of glaucomatous degeneration. Our previous studies identify a detrimental effect of a specific stress response on RGC survival. However, how this stress response contributes to neurodegeneration is unclear. We reasoned that identifying specific downstream effectors and upstream regulators of this specific stress condition is essential for efficient neural repair. We are taking advantage of newly developed genetic tools to survey gene expression and epigenetic regulatory elements that are associated with RGC at normal function, under disease, or after treatment. Through this effort, we will be able to draw a comprehensive gene regulatory network in RGC specifically, which then serve as a blueprint for developing novel and effective neuroprotectants for glaucoma.