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Research in the Ferrington Laboratory is focused on investigating age-related macular degeneration (AMD), the number one cause of blindness among the elderly. There are two clinically distinct forms of the disease, wet AMD and dry AMD. Wet AMD, resulting from abnormal growth of blood vessels into the retina, has several effective treatments available to prevent vision loss. Dry AMD is a progressive disease that culminates in central vision impairment due to the death of the retinal pigment epithelium (RPE) and subsequent loss of the light-sensing photoreceptors in the macula. Currently no treatments are available for dry AMD, which includes more than 80% of AMD patients. Discovering potential targets for therapy and testing promising treatment candidates requires both a clear understanding of the disease mechanism, as well as practical model systems that authentically replicate disease phenotypes.

Our research team has been investigating critical questions driving the field of AMD. What are the cellular changes that occur with aging? What factors “tip the balance” to pathology? How does the cell respond to disease? How can we protect against pathologic changes? To answer these questions, we use tissue and cultured primary retinal pigment epithelial (RPE) cells from human eyebank donors graded for the presence and severity of AMD. We also use RPE differentiated from induced pluripotent stem cells (iPSC-RPE) generated from both eyebank donors and AMD patients. These model systems allow us to investigate the underlying pathophysiology of AMD and to identify drugs that could be used to treat AMD. Our “systems biology” approach includes coupling biochemical analysis of tissues and cells, molecular and cell biology in the Ferrington laboratory, with work of our multidisciplinary team of collaborators who provide complementary expertise in stem cell biology, global protein analysis using label-free mass spectrometry, and targeted metabolomics.