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Gene Ther. 2008 Nov;15(22):1463-8. doi: 10.1038/gt.2008.145. Epub 2008 Sep 11.

Progress and prospects: zinc-finger nucleases as gene therapy agents.

Author information

1
Department of Biochemistry, University of Utah School of Medicine, Salt Lake City, UT 84112-5650, USA. dana@biochem.utah.edu

Abstract

Zinc-finger nucleases (ZFNs) are powerful tools for experimental gene manipulation. A number of recent papers have shown how this technology can be applied effectively to models of human gene therapy. Significant target genes and useful methods of ZFN delivery have been reported. Important strides have been made in minimizing toxic side effects observed with some ZFNs, which bodes well for their ultimate safety. New tools are available for the design and testing of ZFNs for new target genes. Applications of ZFNs to stem cells have been described, and genuine gene therapy trials appear to be on the immediate horizon.

PMID:
18784746
PMCID:
PMC2747807
DOI:
10.1038/gt.2008.145
[Indexed for MEDLINE]
Free PMC Article

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