Posts by Constance Cepko, PhD
Gene Therapy Reverses Degeneration of the Retinal Pigment Epithelium in Preclinical Models
Gene therapy with Nrf2—a master transcription factor—preserves the retinal pigment epithelium in mouse models of retinitis pigmentosa, David M. Wu, MD, PhD, of the Department of Ophthalmology, and colleagues observed.
Connie Cepko is Bullard Professor of Genetics and Neuroscience at Harvard Medical School and a Howard Hughes Medical Institute Investigator. She trained in virology with Phil Sharp at MIT for a Ph.D. and later with Richard Mulligan at the MIT Whitehead Institute. She helped develop retroviral vectors for transduction into the nervous system for lineage analysis and for studies of gene function in vivo. Her laboratory has focused on the topic of cell fate determination in the retina through the analysis of the behavior of progenitor and stem cells. More recently, they have been studying the mechanisms of photoreceptor death in diseases that cause blindness, such as retinitus pigmentosa and macular degeneration.