Recent developments in adeno-associated virus vector technology

被引：137

作者：

Buening, Hildegard ^{[1
,2
]}

Perabo, Luca ^{[1
]}

Coutelle, Oliver ^{[1
]}

Quadt-Humme, Sibille ^{[1
,2
]}

Hallek, Michael ^{[1
,2
]}

机构：

[1] Univ Cologne, Clin Internal Med 1, D-50937 Cologne, Germany

[2] Univ Cologne, Ctr Mol Med Cologne, D-50937 Cologne, Germany

来源：

JOURNAL OF GENE MEDICINE | 2008年 / 10卷 / 07期

关键词：

rAAV; capsid engineering; vector targeting; immune response; gene therapy;

D O I：

10.1002/jgm.1205

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), a single-stranded DNA parvovirus, is emerging as one of the leading gene therapy vectors owing to its nonpathogenicity and low immunogenicity, stability and the potential to integrate site-specifically without known side-effects. A portfolio of recombinant AAV vector types has been developed with the aim of optimizing efficiency, specificity and thereby also the safety of in vitro and in vivo gene transfer. More and more information is now becoming available about the mechanism of AAV/host cell interaction improving the efficacy of recombinant AAV vector (rAAV) mediated gene delivery. This review summarizes the current knowledge of the infectious biology of AAV, provides an overview of the latest developments in the field of AAV vector technology and discusses remaining challenges. Copyright (C) 2008 John Wiley & Sons, Ltd.

引用

页码：717 / 733

页数：17

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