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

Thomas Gaj; Jing Guo; Yoshio Kato; Shannon J. Sirk; Carlos F. Barbas

doi:10.1038/nmeth.2030

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

Thomas Gaj, Jing Guo, Yoshio Kato, Shannon J. Sirk, Carlos F. Barbas

Research output: Contribution to journal › Article › peer-review

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.

Original language	English (US)
Pages (from-to)	805-807
Number of pages	3
Journal	Nature Methods
Volume	9
Issue number	8
DOIs	https://doi.org/10.1038/nmeth.2030
State	Published - Aug 2012
Externally published	Yes

ASJC Scopus subject areas

Biotechnology
Biochemistry
Molecular Biology
Cell Biology

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10.1038/nmeth.2030

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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.",

author = "Thomas Gaj and Jing Guo and Yoshio Kato and Sirk, {Shannon J.} and Barbas, {Carlos F.}",

note = "Funding Information: We thank C. Gersbach for contributing to preliminary studies, P. Ikrenyi for technical assistance and A. Mercer for discussion of the manuscript. This research was supported by US National Institutes of Health (NIH) grants GM065059 and DP1OD006990 and by The Skaggs Institute for Chemical Biology. T.G. was supported by a US National Institute of General Medicine Sciences fellowship (T32GM080209). Y.K. was supported by the Japan Society for the Promotion of Science Research Fellowships for Young Scientists.",

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AU - Barbas, Carlos F.

N1 - Funding Information: We thank C. Gersbach for contributing to preliminary studies, P. Ikrenyi for technical assistance and A. Mercer for discussion of the manuscript. This research was supported by US National Institutes of Health (NIH) grants GM065059 and DP1OD006990 and by The Skaggs Institute for Chemical Biology. T.G. was supported by a US National Institute of General Medicine Sciences fellowship (T32GM080209). Y.K. was supported by the Japan Society for the Promotion of Science Research Fellowships for Young Scientists.

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

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