Age-related macular degeneration (AMD) is the leading cause of substantial and irreversible vision loss amongst elderly populations\nin industrialized countries. The advanced neovascular (or ââ?¬Å?wetââ?¬Â) form of the disease is responsible for severe and aggressive loss of\ncentral vision. Current treatments aim to seal off leaky blood vessels via laser therapy or to suppress vessel leakage and neovascular\ngrowth through intraocular injections of antibodies that target vascular endothelial growth factor (VEGF).However, the long-term\nsuccess of anti-VEGF therapy can be hampered by limitations such as low or variable efficacy, high frequency of administration\n(usually monthly), potentially serious side effects, and, most importantly, loss of efficacy with prolonged treatment. Gene transfer\nof endogenous antiangiogenic proteins is an alternative approach that has the potential to provide long-term suppression of\nneovascularization and/or excessive vascular leakage in the eye. Preclinical studies of gene transfer in a large animal model have\nprovided impressive preliminary results with a number of transgenes. In addition, a clinical trial in patients suffering fromadvanced\nneovascular AMD has provided proof-of-concept for successful gene transfer. In this mini review, we summarize current theories\npertaining to the application of gene therapy for neovascular AMD and the potential benefits when used in conjunction with\nendogenous antiangiogenic proteins.
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