Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success-A Personal Perspective
Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success-A Personal Perspective
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DOI：
10.1089/hum.2015.025
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发表时间：
2015-05-01
影响因子：
4.2
通讯作者：
Samulski, R. Jude
中科院分区：
文献类型：
--
作者：
Hastie, Eric;Samulski, R. Jude
在腺相关病毒（AAV）被发现50年以及首次进行基因转移实验30多年后，数十项基因治疗临床试验正在进行中，一种载体在欧洲被批准使用，并且病毒改造和疾病建模方面的突破正在为罕见病、癌症以及艾滋病治疗的革命铺平道路。这篇综述将从历史角度阐述AAV用于基因治疗从发现到临床的进展，重点关注萨穆尔斯基实验室在AAV基础科学和克隆、载体的优化大规模生产、临床前大型动物研究和安全性数据、为提高疗效进行的载体改造以及成功的临床应用方面所做出的贡献。
Fifty years after the discovery of adeno-associated virus (AAV) and more than 30 years after the first gene transfer experiment was conducted, dozens of gene therapy clinical trials are in progress, one vector is approved for use in Europe, and breakthroughs in virus modification and disease modeling are paving the way for a revolution in the treatment of rare diseases, cancer, as well as HIV. This review will provide a historical perspective on the progression of AAV for gene therapy from discovery to the clinic, focusing on contributions from the Samulski lab regarding basic science and cloning of AAV, optimized large-scale production of vectors, preclinical large animal studies and safety data, vector modifications for improved efficacy, and successful clinical applications.