Drug discovery with engineered zinc-finger proteins

Nat Rev Drug Discov. 2003 May;2(5):361-8. doi: 10.1038/nrd1087.

Abstract

Zinc-finger proteins (ZFPs) that recognize novel DNA sequences are the basis of a powerful technology platform with many uses in drug discovery and therapeutics. These proteins have been used as the DNA-binding domains of novel transcription factors (ZFP TFs), which are useful for validating genes as drug targets and for engineering cell lines for small-molecule screening and protein production. Recently, they have also been used as a basis for novel human therapeutics. Most of our advances in the design and application of these ZFP TFs rely on our ability to engineer ZFPs that bind short stretches of DNA (typically 9-18 base pairs) located within the promoters of target genes. Here, we summarize the methods used to design these DNA-binding domains, explain how they are incorporated into novel transcription factors (and other useful molecules) and describe some key applications in drug discovery.

Publication types

  • Review

MeSH terms

  • Base Sequence
  • Binding Sites
  • DNA / chemistry
  • DNA / metabolism
  • Drug Design*
  • Gene Expression Regulation
  • Humans
  • Protein Engineering*
  • Transcription Factors / metabolism*
  • Zinc Fingers*

Substances

  • Transcription Factors
  • DNA