Terence Flotte

Dr. Terence Flotte is a Professor at the UMass Chan Medical School, holding key roles in the Department of Genetic and Cellular Medicine and affiliations across multiple departments including Microbiology, Pediatrics (Division of Pediatric Pulmonology), and the RNA Therapeutics Institute. His research focuses on gene therapy for genetic diseases affecting children, particularly cystic fibrosis (CF), alpha-1 antitrypsin (AAT) deficiency, and fatty acid oxidation disorders. He pioneered the use of adeno-associated virus (AAV) vectors for in vivo gene transfer, including the first human trials for CFTR gene delivery via AAV.

Dr. Flotte earned his BS in Biological Sciences from the University of New Orleans (1982) and his MD from Louisiana State University Medical Center (1986), followed by pediatric residency and pulmonary fellowship at Johns Hopkins University. His lab’s work on AAV vectors, their integration, persistence, and immune responses has advanced gene therapy for AAT deficiency, Tay-Sachs disease, and fatty oxidation disorders. Recent efforts include optimizing AAV1 and AAV9 serotypes for systemic delivery and exploring hybrid rAAV-RNAi approaches.

His publications span clinical trials, vector engineering, and immune modulation in gene therapy, with a strong emphasis on translating preclinical findings to human applications. Collaborations include studies on liver-directed gene therapy, neurodegenerative diseases, and ethical/science policy challenges in the field.