title: Targeted gene knockout by direct delivery of zinc-finger nuclease proteins.
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.
authors: The Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, California, USA.
authors: Thomas
authors: T
authors: Gaj
authors: Jing
authors: J
authors: Guo
authors: Yoshio
authors: Y
authors: Kato
authors: Shannon J
authors: SJ
authors: Sirk
authors: Carlos F
authors: CF
authors: Barbas
mesh_terms: D020816:Amino Acid Motifs; D000595:Amino Acid Sequence; D000818:Animals; D016466:CHO Cells; D020134:Catalytic Domain; D002463:Cell Membrane Permeability; D006224:Cricetinae; D015252:Deoxyribonucleases, Type II Site-Specific / Q000737:chemistry / Q000378:metabolism*; D055786:Gene Knockout Techniques / Q000379:methods*; D005818:Genetic Engineering / Q000379:methods*; D006801:Humans; D008969:Molecular Sequence Data; D010455:Peptides / Q000737:chemistry; D019713:Receptors, CCR5 / Q000235:genetics; D011993:Recombinant Fusion Proteins / Q000737:chemistry / Q000235:genetics / Q000378:metabolism*; D016335:Zinc Fingers / Q000235:genetics / Q000502:physiology*; D054322:tat Gene Products, Human Immunodeficiency Virus / Q000737:chemistry
chemical_list: D010455:Peptides; D019713:Receptors, CCR5; D011993:Recombinant Fusion Proteins; D054322:tat Gene Products, Human Immunodeficiency Virus; C015462:polyarginine; C035042:endodeoxyribonuclease FokI; D015252:Deoxyribonucleases, Type II Site-Specific