The 'cutting edge' of gene therapy... literally - Zinc-finger exercises

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2 Scopus citations

Abstract

The traditional gene-therapy approach relies on the delivery of a therapeutic transgene into a cell, typically to compensate for a gene that is not functional owing to a genetic defect. But why not just correct the genetic defects directly? The answer used to be that there was no methodology for making precise genetic modifications in a highly efficient manner. That is changing now. Over the last decade, researchers have devised a way to stimulate the natural DNA-repair mechanisms of the cell to occur at any desired site in the genome. The enabling technological advance has been the development of programmable nucleases, which use re-engineered ZF (zinc-finger) DNA-binding domains to cut the DNA in a living cell at a precise user-defined location. These methods have been shown to produce genetic modifications at frequencies of >1 correct event per ten treated cells, representing a 100000-fold stimulation of targeted gene repair. The first Phase I clinical trial of a therapeutic ZFN (zinc-finger nuclease) is scheduled for 2008.

Original languageEnglish (US)
Pages (from-to)10-13
Number of pages4
JournalBiochemist
Volume30
Issue number3
StatePublished - Jun 2008

Keywords

  • Engineered zinc finger
  • Gene repair
  • Gene therapy
  • HIV
  • Homologous recombination
  • Mutation correction
  • Protein engineering
  • Zinc-finger nuclease

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

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