Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

被引：87

作者：

Gaj, Thomas ^{[1
]}

Epstein, Benjamin E. ^{[2
]}

Schaffer, David V. ^{[1
,2
,3
,4
]}

机构：

[1] Univ Calif Berkeley, Dept Chem & Biomol Engn, Berkeley, CA 94720 USA

[2] Univ Calif Berkeley, Dept Bioengn, Berkeley, CA 94720 USA

[3] Univ Calif Berkeley, Dept Cell & Mol Biol, Berkeley, CA 94720 USA

[4] Univ Calif Berkeley, Helen Wills Neurosci Inst, Berkeley, CA 94720 USA

来源：

MOLECULAR THERAPY | 2016年 / 24卷 / 03期

基金：

美国国家卫生研究院;

关键词：

SITE-SPECIFIC INTEGRATION; HEPARAN-SULFATE PROTEOGLYCAN; IN-VIVO; HOMOLOGOUS RECOMBINATION; VECTOR INTEGRATION; GENE-THERAPY; TARGETED INTEGRATION; DNA-SEQUENCES; MOUSE MODEL; STEM-CELLS;

D O I：

10.1038/mt.2015.151

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：

In addition to their broad potential for therapeutic gene delivery, adeno-associated virus (AAV) vectors possess the innate ability to stimulate homologous recombination in mammalian cells at high efficiencies. This process referred to as AAV-mediated gene targeting has enabled the introduction of a diverse array of genomic modifications both in vitro and in vivo. With the recent emergence of targeted nucleases, AAV-mediated genome engineering is poised for clinical translation. Here, we review key properties of AAV vectors that underscore its unique utility in genome editing. We highlight the broad range of genome engineering applications facilitated by this technology and discuss the strong potential for unifying AAV with targeted nucleases for next-generation gene therapy.

引用

页码：458 / 464

页数：7

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