From cell biology to therapy for blinding diseases


The goal of our research is to use cell biology to discover novel therapies for blinding diseases such as age-related macular degeneration (AMD).  This devastating disease, which robs people of central high-resolution vision, affects over 30 million older adults worldwide.  There are currently no effective treatments for the chronic form of AMD called geographic atrophy, which leads to a slow decline in vision in 90% of patients.


Our research focuses on the retinal pigment epithelium (RPE), the site of the initial insult that eventually leads to vision loss in macular degeneration and other retinal diseases.  A key feature of AMD is the accumulation of cellular debris within and around the RPE, which can compromise RPE function and induce inflammation to promote disease.

We use high-speed, high-resolution live imaging of healthy and diseased RPE and genetically engineered animal models to investigate various aspects of RPE physiology including autophagy, lysosome function, organelle biogenesis and transport, mitochondrial dynamics and intercellular communication within the retina.

The image above is a 3-D reconstruction of a sheet of polarized primary RPE showing the recycling system of the cells, comprised of lysosomes in orange and autophagosomes in green. Individual RPE cells are demarcated by tight junctions shown in pale pink and the nuclei are in blue.


Recent research from our laboratory has identified novel pathways that can be targeted by existing FDA-approved drugs to preserve the health and function of the retinal pigment epithelium over a lifetime.  Studies are currently underway to establish the efficacy of these drugs in preventing inflammation in the retina and in preserving vision in models of macular degeneration.

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