Adeno-associated virus vector as a platform for gene therapy delivery

被引：1663

作者：

Wang, Dan ^{[1
,2
,3
]}

Tai, Phillip W. L. ^{[1
,2
,3
]}

Gao, Guangping ^{[1
,2
,3
]}

机构：

[1] Univ Massachusetts, Sch Med, Horae Gene Therapy Ctr, Worcester, MA 01655 USA

[2] Univ Massachusetts, Sch Med, Li Weibo Inst Rare Dis Res, Worcester, MA 01655 USA

[3] Univ Massachusetts, Sch Med, Dept Microbiol & Physiol Syst, Worcester, MA 01655 USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2019年 / 18卷 / 05期

基金：

美国国家卫生研究院;

关键词：

ADENO-ASSOCIATED-VIRUS; CENTRAL-NERVOUS-SYSTEM; BLOOD-BRAIN-BARRIER; POSTTRANSCRIPTIONAL REGULATORY ELEMENT; RECOMBINANT AAV VECTORS; RATE-LIMITING STEP; IN-VIVO; TRANSGENE EXPRESSION; VIRAL VECTORS; MOUSE MODEL;

D O I：

10.1038/s41573-019-0012-9

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：

Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growth of the gene therapy field. Preclinical and clinical successes in AAV-mediated gene replacement, gene silencing and gene editing have helped AAV gain popularity as the ideal therapeutic vector, with two AAV-based therapeutics gaining regulatory approval in Europe or the United States. Continued study of AAV biology and increased understanding of the associated therapeutic challenges and limitations will build the foundation for future clinical success.

引用

页码：358 / 378

页数：21

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