Recombinant adeno-associated virus as delivery vector for gene therapy - A review

被引：46

作者：

Lu, Y ^{[1
]}

机构：

[1] Johns Hopkins Univ, Sch Med, Dept Neurol, Baltimore, MD 21287 USA

来源：

STEM CELLS AND DEVELOPMENT | 2004年 / 13卷 / 01期

关键词：

D O I：

10.1089/154732804773099335

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

Recombinant adeno-associated virus (rAAV) is one of the most promising delivery vectors for gene therapy, due to its nonpathogenic property, nonimmunogenecity to host, and broad cell and tissue tropisms. This article summarizes the biological characteristics of AAV; the procedures to prepare, purify, and characterize the rAAV for gene therapy applications; and some of the clinical trials utilizing rAAV as delivery vehicles. Also discussed are the current efforts to modify rAAV to change its tropism, the application of different promoters to accommodate specific transgene expression, and the strategy to expand its capacity.

引用

页码：133 / 145

页数：13

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