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PMID: 18784746 Published · ppublish English Journal Article Review

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

Gene therapy ·Vol. 15 ·No. 22 ·2008-11-00 ·Pages 1463-8

Carroll D

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.

MeSH Terms
Animals Endonucleases/genetics Forecasting Genetic Engineering/methods Genetic Therapy/methods,trends Genetic Vectors/genetics Humans Zinc Fingers/genetics
Chemicals
Endonucleases
Authors & Affiliations
1 authors, click to expand affiliations / ORCID
Carroll D
Department of Biochemistry, University of Utah School of Medicine, Salt Lake City, UT 84112-5650, USA. [email protected]
References (22)
22 references, click to expand
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Article Info
Journal
Gene therapy
Abbr.
Gene Ther
ISSN
1476-5462
DOI
10.1038/gt.2008.145
Email
[email protected]
Published
2008-11-00
Epub
2008-00-11
Pages
1463-8
Language
English
Region
England
NLM ID
9421525
PMCID
PMC2747807
Subset
IM
Grants
NIGMS NIH HHS · R01 GM078571 · United States
NIGMS NIH HHS · R01 GM078571-01 · United States
NIGMS NIH HHS · R01 GM078571-02 · United States
NIGMS NIH HHS · R01 GM078571-03 · United States
External Links
PubMed source DOI: 10.1038/gt.2008.145 PMC Full Text View journal details
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