Guangping Gao

Guangping Gao, Ph.D., is the Penelope Booth Rockwell Professor in Biomedical Research, Chair and Professor of Microbiology and Physiological Systems, Director of the Horae Gene Therapy Center, and Director of the Li Weibo Institute for Rare Diseases Research at the University of Massachusetts Chan Medical School. Dr. Gao is internationally recognized as the discoverer of AAV serotype 9 (AAV9), one of the most transformative breakthroughs in gene therapy. While working in James Wilson's laboratory at the University of Pennsylvania in 2003, Gao identified AAV9—the first AAV serotype capable of crossing the blood-brain barrier following peripheral intravenous injection. In neonatal mice, AAV9 produced global neuronal transduction throughout the brain and spinal cord after systemic administration, eliminating the need for invasive direct brain injection. AAV9's unique CNS-tropism revolutionized neurogenetic disease treatment. It enabled clinical trials for Canavan disease and ultimately led to FDA approval of onasemnogene abeparvovec (Zolgensma) in 2019 for spinal muscular atrophy—the first one-time treatment addressing the genetic root cause of SMA, representing a milestone in pediatric neurogenetic medicine. In 2011, Gao joined UMass Chan Medical School and founded the Horae Gene Therapy Center (named with the Chinese characters meaning "harmony and hope"), establishing it as an internationally leading AAV research and development institution. His laboratory focuses on AAV vector engineering through rational design and directed evolution strategies to optimize capsid properties, creating AAV variants with enhanced CNS targeting and reduced peripheral tissue off-target effects. Gao's research program remains highly active in 2025: in September, his team used AAV to deliver HIV broadly neutralizing antibodies for prophylactic therapy; in May, he received $500,000 from Origen Biotechnology to advance AAV transduction efficiency. He also partners with ASC Therapeutics to develop novel gene therapy for maple syrup urine disease. As an internationally recognized pioneer who played a key role in discovering and characterizing new AAV serotype families, Dr. Gao's work has been instrumental in reviving and advancing the gene therapy field.

Guangping Gao哲学博士，是马萨诸塞大学陈氏医学院Penelope Booth Rockwell生物医学研究教授、微生物学与生理系统系主任兼教授、和瑞基因治疗中心主任、李伟波罕见病研究所主任。 Gao博士国际公认为AAV血清型9 (AAV9)的发现者，这是基因治疗领域最具变革性的突破之一。2003年在宾夕法尼亚大学James Wilson实验室工作期间，Gao鉴定出AAV9——首个能够在外周静脉注射后穿越血脑屏障的AAV血清型。在新生小鼠中，AAV9在全身给药后在整个大脑和脊髓产生全局神经元转导，消除了侵入性直接脑内注射的需要。 AAV9独特的CNS趋向性革新了神经遗传疾病治疗。它促成了Canavan病临床试验，并最终导致2019年FDA批准onasemnogene abeparvovec (Zolgensma)用于脊髓性肌萎缩症——首个解决SMA遗传根本原因的一次性治疗，代表儿科神经遗传医学的里程碑。 2011年，Gao加入麻省大学陈氏医学院并创建了和瑞基因治疗中心（以中文"和瑞"命名，寓意"和谐与希望"），将其建设成国际领先的AAV研究与开发机构。他的实验室专注于通过理性设计和定向进化策略进行AAV载体工程化，优化衣壳特性，创建具有增强CNS靶向和减少外周组织脱靶效应的AAV变体。 Gao的研究项目在2025年仍非常活跃：9月，他的团队使用AAV递送HIV广谱中和抗体用于预防性治疗；5月，他从Origen Biotechnology获得50万美元以推进AAV转导效率。他还与ASC Therapeutics合作开发枫糖尿病的新型基因治疗。 作为在发现和表征新AAV血清型家族中发挥关键作用的国际公认先驱，Gao博士的工作对复兴和推进基因治疗领域至关重要。