Targeted gene knockout by direct delivery of zinc-finger nuclease proteins.

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins.
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DOI:
10.1038/nmeth.2030
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发表时间:
2012-07-01
期刊:
影响因子:
48
通讯作者:
Barbas, Carlos F., III
Barbas, Carlos F., III
中科院分区:
生物学1区
文献类型:
--
作者:
Gaj, Thomas;Guo, Jing;Kato, Yoshio;Sirk, Shannon J.;Barbas, Carlos F., III

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锌指核酸酶(ZFN)是重新定义基因组工程的通用试剂。实现这项技术的全部潜力需要开发安全有效的方法将ZFN递送到细胞中。我们证明了标准ZFN结构的内在细胞穿透能力,并表明ZFN作为蛋白质的直接递送导致在各种哺乳动物细胞类型中有效的内源性基因破坏,具有最小的脱靶效应。
Zinc-finger nucleases (ZFNs) are versatile reagents that have redefined genome engineering. Realizing the full potential of this technology requires the development of safe and effective methods for delivering ZFNs into cells. We demonstrate the intrinsic cell-penetrating capabilities of the standard ZFN architecture and show that direct delivery of ZFNs as proteins leads to efficient endogenous gene disruption in a variety of mammalian cell types with minimal off-target effects.
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