Identification and Therapeutic Targeting of Pathogenic Nucleic Acids in AMD

Vision loss in developed nations due to age-related macular degeneration (AMD) continues to reach epidemic proportions in the 21st century. Around the turn of the millennium, the disease was already estimated to affect between 30 and 50 million people worldwide. Currently, it is the leading cause of irreversible blindness on three continents and imparts a massive toll on the health-care system. In the United States, it is likely that future expenses will maintain steady, if not exponential, growth in the face of an aging baby boomer generation and limited financial resources. Severe vision loss from AMD results from choroidal neovascularization (CNV), the invasion of the retina by abnormal choroidal blood vessels, or from geographic atrophy (GA), the apoptotic loss of retinal pigmented epithelium (RPE), photoreceptors and choriocapillaris. Significant advances in the molecular understanding of CNV pathogenesis have led to an FDA-approved vision-improving therapy [1, 2]. In contrast, GA pathogenesis is still nebulous and there are no FDA-approved therapies for the 1 million people in the United States who already have GA and the millions more who are at risk. This proposal aims to provide an enriched training environment for the candidate to gain expertise in next generation biomedical technologies, to advance and translate scientific investigations into recently discovered molecular pathways in non-exudative AMD or GA pathogenesis, and to initiate a Phase I clinical trial with a novel medical therapy to arrest the progression of this blinding disease.