Steven T. Bailey, MD Dr. Bailey is currently completing a retina fellowship at the Oregon Health and Science University's Casey Eye Institute in Portland. Bio

J. Timothy Stout, MD, PhD, MBA Dr. Stout is an associate professor of Ophthalmology at Casey Eye Institute in Portland, Oregon. Bio

The authors state that they have no financial relationship with the manufacturer or provider of any product or service discussed in this article or with the manufacturer or provider of any competing product or service.

Familial aggregation studies, segregation analyses, and twin studies have provided strong evidence for a significant genetic role in the etiology of age-related macular degeneration (AMD) (Surv Ophthalmol. 2006;51:313-363). It is unlikely that researchers will pinpoint a single, causative “defective” gene that is responsible for the pathophysiology that leads to choroidal neovascularization (CNV) and/or geographic atrophy in AMD patients. Rather, the disease is likely caused by a complex interaction between a variety of environmental factors and several genes or gene products. The success of monoclonal antibodies and aptamers against vascular endothelial growth factor (VEGF) gene products have supported the concept that there may be a few proteins key to disease progression,^3,4 and this has contributed to the emergence of 2 new approaches toward treating AMD. The first involves the modulation of disease-causing proteins, while the second involves promoting therapeutic gene expression.