Doris Duke Charitable Foundation

Jayakrishna Ambati, M.D.

Biography

Dr. Ambati is Professor of Ophthalmology & Visual Sciences and Professor of Physiology at the University of Kentucky School of Medicine. He serves as Vice Chair of the Department of Ophthalmology & Visual Sciences and holds the Dr. E. Vernon Smith & Eloise C. Smith Endowed Chair in Macular Degeneration Research. Dr. Ambati was born and raised in India, the elder son of Prof. A. Muralimohan Rao, an Indian Institute of Technology-educated mathematician, and Gomathi Rao, a scholar in Tamil literature. He secured his electrical engineering degree at The Johns Hopkins University at the age of 17, and M.D. (magna cum laude) from SUNY Health Science Center at Brooklyn. Following ophthalmology residency at the University of Rochester and retina fellowship at Harvard Medical School, he joined the University of Kentucky in 2001.

Dr. Ambati seeks to identify the molecular mechanisms underlying the development of age-related macular degeneration (AMD), which affects 10 million Americans, and to develop novel therapeutics to prevent and treat this blinding condition. His laboratory has made fundamental discoveries about AMD pathogenesis and the immune pathways that trigger both the atrophic and neovascular forms of this disease. They also discovered an unexpected intersection of small interfering RNA (siRNA) activity, an anti-viral immune receptor, and blood vessel growth that has profound implications for clinical application of siRNA therapeutics. In collaboration with his brother, Dr. Balamurali Ambati, a cornea surgeon and vascular biologist, he answered one of the foremost questions in vascular biology: how does the cornea remain avascular? His laboratory has published several articles in such journals as Nature, Nature Medicine, and the New England Journal of Medicine, and is supported by the National Eye Institute of NIH.

Dr. Ambati is a Clinical Scholar in Translational Research of the Burroughs Wellcome Fund. He received the Physician Scientist Award and the Lew R. Wasserman Merit Award from Research to Prevent Blindness, the Dennis W. Jahnigen Career Development Award from The American Geriatrics Society, and a Career Development Award from The Foundation Fighting Blindness. The University of Kentucky awarded Dr. Ambati the Albert D. and Elizabeth H. Kirwan Memorial Prize and named him a University Research Professor. He is on the Editorial Board of Investigative Ophthalmology & Visual Science and has been elected a member of the American Society for Clinical Investigation. He lives with his wife Kameshwari, an IIT-educated chemist, and their daughters Meenakshi and Vidya Lakshmi in Lexington, Kentucky.

Abstract

Short Non-Interfering RNAs as Novel Therapies for Age-related Macular Degeneration
Age-related macular degeneration (AMD), is the leading of blindness among the elderly in most industrialized nations, and affects as many Americans as all cancers combined. It results in vision loss either through invasion of abnormal blood vessels into the retina (choroidal neovascularization) or via death of retinal cells (geographic atrophy). Dr. Ambati’s laboratory has identified a critical role for toll-like receptor-3 (TLR3), an anti-viral immune receptor that recognized double stranded RNAs, in both geographic atrophy and choroidal neovascularization. The aim of this project is to develop therapeutics for both forms of the disease by either activating or inhibiting TLR3. In tandem, Dr. Ambati seeks to train a new generation of physician-scientists with translational interests in angiogenesis research.