Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success-A Personal Perspective

被引：206

作者：

Hastie, Eric ^{[1
]}

Samulski, R. Jude ^{[1
,2
]}

机构：

[1] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA

[2] Univ N Carolina, Dept Pharmacol, Chapel Hill, NC 27599 USA

来源：

HUMAN GENE THERAPY | 2015年 / 26卷 / 05期

关键词：

ADENO-ASSOCIATED VIRUS; SELF-COMPLEMENTARY VECTORS; IN-VIVO EXPRESSION; HUMAN GAMMA-GLOBIN; RATE-LIMITING STEP; AAV VECTOR; MUSCULAR-DYSTROPHY; INTRAMUSCULAR INJECTION; EFFICIENT TRANSDUCTION; RECOMBINANT PLASMID;

D O I：

10.1089/hum.2015.025

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

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.

引用

页码：257 / 265

页数：9

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