788. Engineered Zinc Finger Protein Transcription Factors as a Potential Therapy for Choroidal Neovascularization

Abstract

Choroidal neovascularization (CNV) is the most common cause of severe vision loss in patients with age-related macular degeneration (AMD). Anti-angiogenesis therapies, especially those that target Vascular Endothelial Growth Factor (VEGF), have shown promise in treating neovascular AMD. However, targeting VEGF alone maynot yield maximal therapeutic effects. Zinc-finger protein transcription factors (ZFP TF) can be engineered to regulate the expression of virtually any endogenous gene. We sought to test the effects in vitro and in vivo ZFPs designed to repress the transcription of VEGF-A and those designed to activate the transcription of Pigment Epithelium Derived Factor (PEDF), a potent anti-angiogenic factor.