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Gene Therapy / 基因治疗AAV Vectors

Guangping Gao

高光平

Ph.D.

🏢University of Massachusetts Chan Medical School(马萨诸塞大学陈医学院)🌐USA

Director, Horae Gene Therapy CenterHorae基因治疗中心主任

2
Key Papers
2
Awards
2
Key Contributions

👥Biography 个人简介

Guangping Gao is a world-leading expert in adeno-associated virus (AAV) biology and gene therapy vector development. His laboratory has discovered hundreds of novel AAV variants from human and non-human primate tissues, significantly expanding the AAV vector toolkit. Gao's work on AAV9 has been particularly transformative, enabling systemic delivery to the central nervous system and leading to the first FDA-approved gene therapy for spinal muscular atrophy (Zolgensma).

高光平是AAV生物学和基因治疗载体开发领域的世界顶级专家。他的实验室从人类和非人灵长类动物组织中发现了数百种新型AAV变体,显著扩展了AAV载体工具包。高光平在AAV9方面的工作尤为具有变革性,使系统性递送到中枢神经系统成为可能,并促成了首个FDA批准的脊髓性肌萎缩症基因治疗药物(Zolgensma)。

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🧪Research Fields 研究领域

AAV BiologyAAV生物学
Gene Therapy Vectors基因治疗载体
CNS Gene Delivery中枢神经系统基因递送

🎓Key Contributions 主要贡献

AAV Discovery and Characterization

Discovered and characterized over 100 novel AAV serotypes, including AAV9 which can cross the blood-brain barrier.

Clinical Translation

Vector development work directly contributed to FDA approval of Zolgensma for SMA and multiple ongoing clinical trials.

Representative Works 代表性著作

[1]

Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues

Journal of Virology (2004)

Systematic characterization of AAV diversity in human tissues, foundation for novel vector development.

[2]

Novel adeno-associated virus capsid improves systemic delivery of AAV-delivered gene therapy

Nature Medicine (2015)

Engineered AAV variants with enhanced transduction efficiency for therapeutic applications.

🏆Awards & Recognition 奖项与荣誉

🏆ASGCT Outstanding Achievement Award (2020)
🏆Horae Gene Therapy Center Founding Director

📄Data Sources 数据来源

Last updated: 2026-03-04 | All information from publicly available academic sources

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