Dr. Bennett’s laboratory studies the molecular genetics of inherited retinal degenerative diseases with the idea of using this knowledge to develop rational approaches for treatment of these diseases. Over the last several years, Dr. Bennett has used the knowledge gained from studying Choroideremia to develop a gene replacement therapy aimed at stopping the progression of the disease. They use a deactivated virus called AAV-2 as their vector – or transport mechanism – to deliver a healthy copy of the Choroideremia gene into affected cells of the retina. Human clinical trials for this Choroideremia gene therapy treatment, the first to be tested in the United States, began in January 2015. Dr. Bennett’s laboratory has developed a similar gene therapy to treat another retinal disease called Leber’s Congenital Amaurosis, which is currently in being tested in phase 3 clinical trials – following positive results in the phase 1/2 trials.