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Literature DB >> 18784746

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

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.

Entities: CellLine Chemical Disease Gene Species

Mesh：

Substances：
Endonucleases

Year: 2008 PMID： 18784746 PMCID： PMC2747807 DOI： 10.1038/gt.2008.145

Source DB: PubMed Journal: Gene Ther ISSN： 0969-7128 Impact factor: 5.250

22 in total

1. Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells.

Authors: Jason Morton; M Wayne Davis; Erik M Jorgensen; Dana Carroll
Journal: Proc Natl Acad Sci U S A Date: 2006-10-23 Impact factor: 11.205

2. Standardized reagents and protocols for engineering zinc finger nucleases by modular assembly.

Authors: David A Wright; Stacey Thibodeau-Beganny; Jeffry D Sander; Ronnie J Winfrey; Andrew S Hirsh; Magdalena Eichtinger; Fengli Fu; Matthew H Porteus; Drena Dobbs; Daniel F Voytas; J Keith Joung
Journal: Nat Protoc Date: 2006 Impact factor: 13.491

3. Design, construction and in vitro testing of zinc finger nucleases.

Authors: Dana Carroll; J Jason Morton; Kelly J Beumer; David J Segal
Journal: Nat Protoc Date: 2006 Impact factor: 13.491

4. Unexpected failure rates for modular assembly of engineered zinc fingers.

Authors: Cherie L Ramirez; Jonathan E Foley; David A Wright; Felix Müller-Lerch; Shamim H Rahman; Tatjana I Cornu; Ronnie J Winfrey; Jeffry D Sander; Fengli Fu; Jeffrey A Townsend; Toni Cathomen; Daniel F Voytas; J Keith Joung
Journal: Nat Methods Date: 2008-05 Impact factor: 28.547

5. Mammalian gene targeting with designed zinc finger nucleases.

Authors: Matthew H Porteus
Journal: Mol Ther Date: 2005-09-19 Impact factor: 11.454

Review 6. Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy.

Authors: Frédéric Pâques; Philippe Duchateau
Journal: Curr Gene Ther Date: 2007-02 Impact factor: 4.391

Review 7. The phiC31 integrase system for gene therapy.

Authors: Michele P Calos
Journal: Curr Gene Ther Date: 2006-12 Impact factor: 4.391

8. Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases.

Authors: Erica A Moehle; E A Moehle; Jeremy M Rock; J M Rock; Ya-Li Lee; Y L Lee; Yann Jouvenot; Y Jouvenot; Russell C DeKelver; R C Dekelver; Philip D Gregory; P D Gregory; Fyodor D Urnov; F D Urnov; Michael C Holmes; M C Holmes
Journal: Proc Natl Acad Sci U S A Date: 2007-02-20 Impact factor: 11.205

9. Zinc Finger Tools: custom DNA-binding domains for transcription factors and nucleases.

Authors: Jeffrey G Mandell; Carlos F Barbas
Journal: Nucleic Acids Res Date: 2006-07-01 Impact factor: 16.971

10. Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases.

Authors: Yannick Doyon; Jasmine M McCammon; Jeffrey C Miller; Farhoud Faraji; Catherine Ngo; George E Katibah; Rainier Amora; Toby D Hocking; Lei Zhang; Edward J Rebar; Philip D Gregory; Fyodor D Urnov; Sharon L Amacher
Journal: Nat Biotechnol Date: 2008-05-25 Impact factor: 54.908

95 in total

Review 1. Zinc-finger nucleases for somatic gene therapy: the next frontier.

Authors: Shamim H Rahman; Morgan L Maeder; J Keith Joung; Toni Cathomen
Journal: Hum Gene Ther Date: 2011-07-22 Impact factor: 5.695

2. Re-programming DNA-binding specificity in zinc finger proteins for targeting unique address in a genome.

Authors: Abhinav Grover; Akshay Pande; Krishna Choudhary; Kriti Gupta; Durai Sundar
Journal: Syst Synth Biol Date: 2011-02-19

Review 3. Genome editing with engineered zinc finger nucleases.

Authors: Fyodor D Urnov; Edward J Rebar; Michael C Holmes; H Steve Zhang; Philip D Gregory
Journal: Nat Rev Genet Date: 2010-09 Impact factor: 53.242

4. Targeting DNA double-strand breaks with TAL effector nucleases.

Authors: Michelle Christian; Tomas Cermak; Erin L Doyle; Clarice Schmidt; Feng Zhang; Aaron Hummel; Adam J Bogdanove; Daniel F Voytas
Journal: Genetics Date: 2010-07-26 Impact factor: 4.562

5. Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome.

Authors: Russell C DeKelver; Vivian M Choi; Erica A Moehle; David E Paschon; Dirk Hockemeyer; Sebastiaan H Meijsing; Yasemin Sancak; Xiaoxia Cui; Eveline J Steine; Jeffrey C Miller; Phillip Tam; Victor V Bartsevich; Xiangdong Meng; Igor Rupniewski; Sunita M Gopalan; Helena C Sun; Kathleen J Pitz; Jeremy M Rock; Lei Zhang; Gregory D Davis; Edward J Rebar; Iain M Cheeseman; Keith R Yamamoto; David M Sabatini; Rudolf Jaenisch; Philip D Gregory; Fyodor D Urnov
Journal: Genome Res Date: 2010-05-27 Impact factor: 9.043