Discovery is an everyday occurrence in the lab of Dr. Jayakrishna Ambati, professor of Physiology and professor and vice-chair in the Department of Ophthalmology and Visual Sciences at the University of Kentucky College of Medicine. Ambati has devoted his life's work to making blindness due to age related macular degeneration (AMD) - the leading cause of blindness in hundreds of thousands of people each year - a thing of the past. His discoveries have moved us closer than ever before to a cure for this disease.
Ambati, an international authority on AMD, has made significant advances toward finding treatments for AMD. Since coming to UK in 2001, Ambati has assembled a team of top-tier scientists and clinicians from around the world to help solve the puzzle of macular degeneration. Findings from Ambati's lab have been published in prestigious scientific journals including: Nature, Cell, Nature Medicine, The Journal of Clinical Investigation, and The Proceedings of the National Academy of Sciences.
Major milestones from the Ambati Lab include:
- Discovery of the first animal model of age-related macular degenerationThe discovery led to a scientifically influential paper published in 2003 in Nature Medicine.
- Discovery of Soluble Vascular Endothelial Growth Factor Receptor 1 (sVEGFR1), a protein that prevents blood vessels from growing into the cornea of the eye The finding was published in 2006 in Nature and hailed as "Breakthrough of the Year" by Science.
- Discovery of how siRNA (Small Interfering RiboNucleic Acid) drugs, which were widely believed to work via the mechanism of RNA interference, were generically stopping blood vessel growth by triggering an immune response even without entering cells or triggering RNA interference. This blockbuster study may have saved the vision of people who could have otherwise lost vision if exposed to the drugs of the first studies, and helped guide the creation of safer "2nd generation" siRNA drugs. This study was published in 2008 in Nature and featured in the New York Times.
- Discovery of why cells in the retina die in the "dry" form of AMD. Ambati's lab identified an enzyme called DICER1 that is deficient in the eyes of people with the advanced form of dry AMD. As the levels of DICER1 go down, a toxin called Alu RNA accumulates and activates a molecular complex known as the inflammasome, which in turn causes cell death in the retina. These findings were published in 2011 in Nature and in April 2012 in Cell.