» Articles » PMID: 22751204

Targeted Gene Knockout by Direct Delivery of Zinc-finger Nuclease Proteins

Overview
Journal Nat Methods
Date 2012 Jul 4
PMID 22751204
Citations 134
Authors
Affiliations
Soon will be listed here.
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.

Citing Articles

Direct protein delivery into intact Arabidopsis cells for genome engineering.

Furuhata Y, Kimura M, Sakai A, Murakami T, Egi E, Sakuma T Sci Rep. 2024; 14(1):22568.

PMID: 39343787 PMC: 11439911. DOI: 10.1038/s41598-024-72978-9.


Nanoparticle-mediated delivery of non-viral gene editing technology to the brain.

Williams L, Larsen J Prog Neurobiol. 2023; 232:102547.

PMID: 38042249 PMC: 10872436. DOI: 10.1016/j.pneurobio.2023.102547.


Researching the future: scenarios to explore the future of human genome editing.

Selin C, Lambert L, Morain S, Nelson J, Barlevy D, Farooque M BMC Med Ethics. 2023; 24(1):72.

PMID: 37735670 PMC: 10512597. DOI: 10.1186/s12910-023-00951-8.


AAV-based in vivo gene therapy for neurological disorders.

Ling Q, Herstine J, Bradbury A, Gray S Nat Rev Drug Discov. 2023; 22(10):789-806.

PMID: 37658167 DOI: 10.1038/s41573-023-00766-7.


Targeted DNA Demethylation: Vectors, Effectors and Perspectives.

Yano N, Fedulov A Biomedicines. 2023; 11(5).

PMID: 37239005 PMC: 10215725. DOI: 10.3390/biomedicines11051334.


References
1.
Pruett-Miller S, Reading D, Porter S, Porteus M . Attenuation of zinc finger nuclease toxicity by small-molecule regulation of protein levels. PLoS Genet. 2009; 5(2):e1000376. PMC: 2633050. DOI: 10.1371/journal.pgen.1000376. View

2.
Guschin D, Waite A, Katibah G, Miller J, Holmes M, Rebar E . A rapid and general assay for monitoring endogenous gene modification. Methods Mol Biol. 2010; 649:247-56. DOI: 10.1007/978-1-60761-753-2_15. View

3.
Hockemeyer D, Soldner F, Beard C, Gao Q, Mitalipova M, DeKelver R . Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases. Nat Biotechnol. 2009; 27(9):851-7. PMC: 4142824. DOI: 10.1038/nbt.1562. View

4.
Holt N, Wang J, Kim K, Friedman G, Wang X, Taupin V . Human hematopoietic stem/progenitor cells modified by zinc-finger nucleases targeted to CCR5 control HIV-1 in vivo. Nat Biotechnol. 2010; 28(8):839-47. PMC: 3080757. DOI: 10.1038/nbt.1663. View

5.
Gordley R, Gersbach C, Barbas 3rd C . Synthesis of programmable integrases. Proc Natl Acad Sci U S A. 2009; 106(13):5053-8. PMC: 2654808. DOI: 10.1073/pnas.0812502106. View