Review

Gene Therapy (2008) 15, 1463–1468; doi:10.1038/gt.2008.145; published online 11 September 2008

Progress and prospects: Zinc-finger nucleases as gene therapy agents

D Carroll1

1Department of Biochemistry, University of Utah School of Medicine, Salt Lake City, UT, USA

Correspondence: Dr D Carroll, Department of Biochemistry, University of Utah School of Medicine, 15 N. Medical Drive East, Room 4100, Salt Lake City, UT 84112-5650, USA. E-mail: dana@biochem.utah.edu

Received 31 July 2008; Revised 26 August 2008; Accepted 27 August 2008; Published online 11 September 2008.

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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.

Keywords:

zinc-finger nucleases, gene targeting, nonhomologous end joining, homologous recombination

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