Progress and prospects: zinc-finger nucleases as gene therapy agents.
Progress and prospects: zinc-finger nucleases as gene therapy agents.
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作者：
锌指核酸酶(ZFN)是实验基因操作的有力工具。最近的一些论文表明，这项技术如何有效地应用于人类基因治疗的模型。ZFN的重要靶基因和有用的传递方法已有报道。一些ZFN在尽量减少毒副作用方面取得了重要进展，这对它们的最终安全性来说是个好兆头。新的工具可用于设计和测试针对新靶基因的ZFN。ZFN在干细胞中的应用已经被描述，真正的基因治疗试验似乎即将到来。
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.
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影响因子：
14.8
作者：
Wright, David A.;Thibodeau-Beganny, Stacey;Joung, J. Keith
通讯作者：
Joung, J. Keith
DOI：
10.1073/pnas.0800940105
发表时间：
2008-04-15
影响因子：
11.1
作者：
Santiago, Yolanda;Chan, Edmond;Collingwood, Trevor N.
通讯作者：
Collingwood, Trevor N.
影响因子：
12.4
作者：
Cornu, Tatjana I.;Thibodeau-Beganny, Stacey;Cathomen, Toni
通讯作者：
Cathomen, Toni
影响因子：
12.4
作者：
Pruett-Miller, Shondra M.;Connelly, Jon P.;Porteus, Matthew H.
通讯作者：
Porteus, Matthew H.
影响因子：
46.9
作者：
Szczepek, Michal;Brondani, Vincent;Cathomen, Toni
通讯作者：
Cathomen, Toni