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

Academic Article
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Overview

related to degree

  • Gaj, Thomas, Ph.D. in Chemical Biology, Scripps Research 2007 - 2013
  • Guo, Jing, Ph.D. in Biology, Scripps Research 2003 - 2010

authors

  • Gaj, Thomas
  • Guo, Jing
  • Kato, Y.
  • Sirk, S. J.
  • Barbas III, Carlos

publication date

  • 2012

journal

  • Nature Methods  Journal

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.

subject areas

  • Amino Acid Motifs
  • Amino Acid Sequence
  • Animals
  • CHO Cells
  • Catalytic Domain
  • Cell Membrane Permeability
  • Cricetinae
  • Deoxyribonucleases, Type II Site-Specific
  • Gene Knockout Techniques
  • Genetic Engineering
  • Humans
  • Molecular Sequence Data
  • Peptides
  • Receptors, CCR5
  • Recombinant Fusion Proteins
  • Zinc Fingers
  • tat Gene Products, Human Immunodeficiency Virus
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Identity

PubMed Central ID

  • PMC3424280

International Standard Serial Number (ISSN)

  • 1548-7091

Digital Object Identifier (DOI)

  • 10.1038/nmeth.2030

PubMed ID

  • 22751204
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Additional Document Info

start page

  • 805

end page

  • 7

volume

  • 9

issue

  • 8

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