Terence R. Flotte, MD, the Celia and Isaac Haidak Professor, is provost and executive deputy chancellor of UMass Chan Medical School and dean of the T.H. Chan School of Medicine, and Vice President of the American Society of Gene and Cell Therapy. The Flotte laboratory has focused on the delivery of therapeutic genes and miRNA with recombinant AAV vectors for the treatment of genetic diseases, such as alpha-1 antitrypsin (AAT) deficiency, Tay-Sachs disease, and cystic fibrosis (CF). In 1995, his lab used rAAV in humans for the first time, in a CF clinical trial on which he was Principal Investigator. His leadership roles as Director of the University of Florida Gene Therapy Center and Genetics Institute, Chair of Pediatrics at UF, and Dean at UMMS have expanded this theme, seeking to create new collaborative communities of physicians and scientists aligned in effective ways to use the exponentially growing body of scientific knowledge to advance the mission of improving patient care. The most recent research effort has included first-in-human trials in Tay-Sachs, preclinical gene editing for antitrypsin deficiency, and studies of immune responses to AAV in humans.
