Terence R. Flotte

Terence R. Flotte, MD is a Professor at UMass Chan Medical School and the Principal Investigator of the Flotte Lab for Gene Therapy at the Horae Gene Therapy Center. His research focuses on developing gene therapy approaches for genetic diseases affecting children, with particular emphasis on alpha-1 antitrypsin deficiency, cystic fibrosis, and fatty acid oxidation disorders.

Dr. Flotte's research interests include:

• Adeno-associated virus (AAV) vector development for gene therapy
• Gene therapy for alpha-1 antitrypsin deficiency
• Cystic fibrosis gene therapy
• Gene therapy for fatty acid oxidation disorders
• Central nervous system gene therapy
• Gene editing approaches using CRISPR/Cas9

Dr. Flotte's recent publications demonstrate a strong focus on translational gene therapy research, with multiple clinical trials underway for lysosomal storage disorders like GM2 gangliosidosis and GM1 gangliosidosis. His work spans from basic vector engineering to clinical applications, with particular attention to delivery methods, immune responses, and safety considerations. Recent publications highlight advances in base editing for alpha-1 antitrypsin deficiency, novel delivery methods including robot-assisted intrathalamic infusion, and detailed characterization of immune responses to AAV vectors.

Dr. Flotte has contributed significantly to the field through numerous reviews and editorials that provide historical perspective on gene therapy development and discuss current challenges and future directions.