Terence R. Flotte

Terence R. Flotte, M.D., is Provost/Dean and Executive Deputy Chancellor of University of Massachusetts Chan Medical School, where he holds the Celia and Isaac Haidak Professor chair with faculty appointments in the Department of Pediatrics and Horae Gene Therapy Center. He serves as Editor-in-Chief of Human Gene Therapy, the field's oldest journal family, since 2015. Dr. Flotte is internationally recognized as a pioneer of human AAV gene therapy. As a pediatric pulmonologist, he and his colleagues at Johns Hopkins became the first to use adeno-associated virus (AAV) as a vehicle to deliver corrective genes to targeted sites in the body. In 1995, Flotte initiated the first-ever human use of AAV vectors in a clinical trial for cystic fibrosis patients—a landmark event in gene therapy history. His 1993 Proceedings of the National Academy of Sciences publication demonstrated stable in vivo expression of the cystic fibrosis transmembrane conductance regulator (CFTR) using AAV vectors, providing the scientific foundation for this groundbreaking trial. Flotte's research program has focused on gene therapy for inherited lung diseases, particularly alpha-1 antitrypsin deficiency (AAT) and cystic fibrosis. His laboratory has conducted multiple Phase 1 and Phase 2 clinical trials for AAT and CF, developing investigational gene therapy products including rAAV2-AAT and rAAV1-AAT for gene augmentation therapy. His 2011 Human Gene Therapy publication reported interim results from a Phase 2 trial demonstrating dose-related AAT expression persisting for years after single intramuscular injection. The 2017 Molecular Therapy paper documented 5-year sustained expression and neutrophil defect repair—one of the longest follow-up studies in AAV gene therapy. Flotte's research has been continuously NIH-funded since 1993. As UMass Chan's chief academic and administrative officer, he oversees all academic activities of basic and clinical science departments. He founded and serves as Scientific Advisor to Applied Genetics Technologies Corporation (AGTC) and serves on the American Society of Gene & Cell Therapy leadership team, shaping policies and standards for the gene therapy field.

Terence R. Flotte医学博士，是马萨诸塞大学陈氏医学院教务长/院长兼执行副校长，担任Celia和Isaac Haidak教授职位，并在儿科系和和瑞基因治疗中心任职。自2015年起担任该领域最古老期刊家族Human Gene Therapy的主编。 Flotte博士国际公认为人类AAV基因治疗的先驱。作为儿科肺科医师，他和约翰霍普金斯大学的同事成为首批使用腺相关病毒(AAV)作为载体将矫正基因递送到体内靶点的人。1995年，Flotte在囊性纤维化患者中启动了首次人类AAV载体使用的临床试验——基因治疗历史上的里程碑事件。他1993年发表在《美国国家科学院院刊》上的论文证明了使用AAV载体稳定体内表达囊性纤维化跨膜转导调节因子(CFTR)，为这一开创性试验提供了科学基础。 Flotte的研究项目专注于遗传性肺病基因治疗，特别是Alpha-1抗胰蛋白酶缺乏症(AAT)和囊性纤维化。他的实验室已进行了多项AAT和CF的I期和II期临床试验，开发了包括rAAV2-AAT和rAAV1-AAT在内的研究性基因治疗产品用于基因增强治疗。他2011年在Human Gene Therapy上发表的论文报告了II期试验的中期结果，证明单次肌肉注射后AAT表达持续数年的剂量相关性。2017年Molecular Therapy论文记录了5年持续表达和中性粒细胞缺陷修复——AAV基因治疗领域最长随访研究之一。 Flotte的研究自1993年以来一直获得NIH资助。作为麻省大学陈氏医学院的首席学术和行政官员，他监督基础和临床科学系的所有学术活动。他创建并担任Applied Genetics Technologies Corporation (AGTC)的科学顾问，并担任美国基因与细胞治疗学会领导团队成员，为基因治疗领域制定政策和标准。