Gene Therapy for Neurodegeneration in Glaucoma

Glaucoma is a leading cause of irreversible visual impairment and blindness worldwide. Although it has been well established that neurodegeneration plays a key role in the disease, current therapies are limited to the modification of risk factors such as elevated intraocular eye pressure. As a novel treatment approach, gene therapy has the promising prospect for addressing genetic changes or for inducing the expression of neuroprotective molecules. This chapter will highlight recent research surrounding these topics and their potential for translational clinical applications within the context of glaucoma subtypes, possible clinical delivery routes, challenges that are being addressed, recent discoveries in glaucoma genetics, and identification of biochemical pathways that attenuate neuronal cell death. Although more basic science and preclinical work are required before possible clinical trials, the intrinsic characteristics of human eye anatomy and current clinical practices enhance the feasibility and promise of using gene therapy to treat neurodegeneration in glaucoma.