Regulation and Translation of Human Gene Therapy

Gene Therapy holds the promise to treat many genetic conditions for which there is no cure. Dr. Gao, who has been working in the field of human gene therapy for the last 30 years, gave a wonderful perspective of where the field stands, which are the challenges ahead, and how they can be overcome. By talking about challenges he faced in of his own research on Canavan Disease and Maple Syrup Urine Disease, he explained how better and improved regulation of gene expression can be achieved for a more physiological outcome and a more successful gene therapy. His seminar was an inspiration for all ophthalmology trainees in the audience. More on Dr. Gao’s research can be found on his UMass webpage.