RNA Interference in model systems of macular degeneration.

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Exudative age-related macular degeneration (AMD) is the most common cause of irreversible severe vision loss in the elderly, with an estimated 25-30 million people worldwide blind due to AMD. There is currently no standard treatment for AMD. A safe, specific and effective pharmacologic agent for AMD, therefore, has enormous therapeutic, social and economic benefits. AMD is underpinned by vascular leakiness and new blood vessel formation. We have demonstrated that Egr-1, a nuclear transcription factor, plays a key regulatory role in a diverse array of angiogenic processes. In this project, we will use novel gene-targeting agents (Egr-1 siRNA) to provide preclinical proof-of principle evidence of their therapeutic potential in established animal models of AMD. These studies will pre-empt Phase IA safety trials in AMD patients.