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Gene Therapy / 基因治疗AAV Vectors

Federico Mingozzi

Ph.D.

🏢Spark Therapeutics / University of Paris🌐USA/France

Chief Scientific Officer

2
Key Papers
2
Key Contributions

👥Biography 个人简介

Federico Mingozzi is an expert in AAV immunology and has made fundamental contributions to understanding immune responses to gene therapy vectors. His research has been instrumental in developing strategies to overcome pre-existing immunity to AAV and managing immune responses in clinical trials. Mingozzi's work has been particularly impactful for liver-directed gene therapy in hemophilia, contributing to the development of approved gene therapies.

Federico Mingozzi是AAV免疫学专家,在理解基因治疗载体免疫反应方面做出了基础性贡献。他的研究在开发克服AAV预存免疫力和管理临床试验免疫反应策略方面发挥了重要作用。Mingozzi的工作对血友病肝脏定向基因治疗产生了特别重要的影响,促进了已获批基因治疗药物的开发。

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🧪Research Fields 研究领域

AAV ImmunologyAAV免疫学
Hemophilia Gene Therapy血友病基因治疗
Liver-Directed Gene Therapy肝脏定向基因治疗

🎓Key Contributions 主要贡献

AAV Immunology

Characterized T cell responses to AAV capsid antigens in clinical trials, leading to immunomodulation strategies.

Hemophilia Gene Therapy

Key contributions to preclinical and clinical development of liver-directed factor VIII and IX gene therapy.

Representative Works 代表性著作

[1]

CD8+ T-cell responses to adeno-associated virus capsid in humans

Nature Medicine (2007)

First demonstration that T cell responses to AAV capsid can limit gene therapy efficacy in humans.

[2]

Immune responses to AAV in clinical trials

Current Gene Therapy (2011)

Comprehensive review of immune challenges in AAV gene therapy clinical development.

📄Data Sources 数据来源

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

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