Progress and prospects: zinc-finger nucleases as gene therapy agents.
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.
Carroll
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Department of Biochemistry, University of Utah School of Medicine, Salt Lake City, UT 84112-5650, USA. dana@biochem.utah.edu
D000818:Animals; D004720:Endonucleases / Q000235:genetics*; D005544:Forecasting; D005818:Genetic Engineering / Q000379:methods; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000235:genetics*; D006801:Humans; D016335:Zinc Fingers / Q000235:genetics*
D004720:Endonucleases
Nat Protoc. 2006;1(3):1637-52
17406455
Nat Biotechnol. 2007 Jul;25(7):778-85
17603475
Curr Gene Ther. 2006 Dec;6(6):633-45
17168696
Nat Biotechnol. 2007 Nov;25(11):1298-306
17965707
Nat Protoc. 2006;1(3):1329-41
17406419
Nat Biotechnol. 2007 Jul;25(7):786-93
17603476
Nucleic Acids Res. 2006 Jul 1;34(Web Server issue):W516-23
16845061
Mol Ther. 2008 Jul;16(7):1200-7
18545224
Mol Ther. 2006 Feb;13(2):438-46
16169774
Nat Biotechnol. 2008 Jun;26(6):695-701
18500337
Nat Biotechnol. 2008 Jul;26(7):808-16
18587387
Proc Natl Acad Sci U S A. 2006 Oct 31;103(44):16370-5
17060623
Nat Biotechnol. 2008 Jun;26(6):702-8
18500334
Genetics. 2006 Apr;172(4):2391-403
16452139
Mol Cell. 2008 Jul 25;31(2):294-301
18657511
Curr Gene Ther. 2007 Feb;7(1):49-66
17305528
Nat Methods. 2008 May;5(5):374-5
18446154
Proc Natl Acad Sci U S A. 2008 Apr 15;105(15):5809-14
18359850
Proc Natl Acad Sci U S A. 2007 Feb 27;104(9):3055-60
17360608
Mol Ther. 2008 Apr;16(4):707-17
18334988
Nucleic Acids Res. 2008 Jul;36(12):3926-38
18511461
Mol Ther. 2008 Feb;16(2):352-358
28178540