Targeted gene knockout by direct delivery of zinc-finger nuclease proteins |
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Authors: | Gaj Thomas Guo Jing Kato Yoshio Sirk Shannon J Barbas Carlos F |
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Institution: | The Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, California, USA. |
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Abstract: | 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 various mammalian cell types with minimal off-target effects. |
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