Recombinant adeno-associated virus vectors for gene therapy

被引：36

作者：

Conlon, TJ ^{[1
]}

Flotte, TR ^{[1
]}

机构：

[1] Univ Florida, Coll Med, Dept Pediat, Gainesville, FL 32610 USA

来源：

EXPERT OPINION ON BIOLOGICAL THERAPY | 2004年 / 4卷 / 07期

关键词：

adeno-associated virus; gene therapy; viral vectors;

D O I：

10.1517/14712598.4.7.1093

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：

Recombinant adeno-associated virus (rAAV) vectors are based on a non-pathogenic human parvovirus (AAV) that is unique in its ability to persist in human cells without causing any pathologic effects. Studies of the potential barriers to rAAV-mediated transduction of relatively resistant cells has led to an understanding of the mechanisms of cell attachment and entry, cytoplasmic translocation, nuclear entry, conversion to active double-stranded DNA activation of transcription and establishment of persistent molecular forms. Each of these areas is individually discussed, as are recent applications in vivo in preclinical models and clinical trials.

引用

页码：1093 / 1101

页数：9

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