Progress and prospects: Zinc-finger nucleases as gene therapy agents

被引：168

作者：

Carroll, D. ^{[1
]}

机构：

[1] Univ Utah, Sch Med, Dept Biochem, Salt Lake City, UT 84112 USA

来源：

GENE THERAPY | 2008年 / 15卷 / 22期

关键词：

zinc-finger nucleases; gene targeting; nonhomologous end joining; homologous recombination;

D O I：

10.1038/gt.2008.145

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Zinc-finger nucleases (ZFNs) are powerful tools for experimental gene manipulation. A number of recent papers have shown how this technology can be applied effectively to models of human gene therapy. Significant target genes and useful methods of ZFN delivery have been reported. Important strides have been made in minimizing toxic side effects observed with some ZFNs, which bodes well for their ultimate safety. New tools are available for the design and testing of ZFNs for new target genes. Applications of ZFNs to stem cells have been described, and genuine gene therapy trials appear to be on the immediate horizon.

引用

页码：1463 / 1468

页数：6

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