An evolutionarily conserved synthetic lethal interaction network identifies FEN1 as a broad-spectrum target for anticancer therapeutic development

PLoS Genet. 2013;9(1):e1003254. doi: 10.1371/journal.pgen.1003254. Epub 2013 Jan 31.

Abstract

Harnessing genetic differences between cancerous and noncancerous cells offers a strategy for the development of new therapies. Extrapolating from yeast genetic interaction data, we used cultured human cells and siRNA to construct and evaluate a synthetic lethal interaction network comprised of chromosome instability (CIN) genes that are frequently mutated in colorectal cancer. A small number of genes in this network were found to have synthetic lethal interactions with a large number of cancer CIN genes; these genes are thus attractive targets for anticancer therapeutic development. The protein product of one highly connected gene, the flap endonuclease FEN1, was used as a target for small-molecule inhibitor screening using a newly developed fluorescence-based assay for enzyme activity. Thirteen initial hits identified through in vitro biochemical screening were tested in cells, and it was found that two compounds could selectively inhibit the proliferation of cultured cancer cells carrying inactivating mutations in CDC4, a gene frequently mutated in a variety of cancers. Inhibition of flap endonuclease activity was also found to recapitulate a genetic interaction between FEN1 and MRE11A, another gene frequently mutated in colorectal cancers, and to lead to increased endogenous DNA damage. These chemical-genetic interactions in mammalian cells validate evolutionarily conserved synthetic lethal interactions and demonstrate that a cross-species candidate gene approach is successful in identifying small-molecule inhibitors that prove effective in a cell-based cancer model.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biological Evolution
  • Cell Proliferation
  • Chromosomal Instability*
  • Colorectal Neoplasms / drug therapy
  • Colorectal Neoplasms / genetics*
  • Colorectal Neoplasms / metabolism
  • DNA Damage
  • DNA-Binding Proteins / genetics
  • DNA-Binding Proteins / metabolism
  • Flap Endonucleases* / antagonists & inhibitors
  • Flap Endonucleases* / genetics
  • Flap Endonucleases* / metabolism
  • Gene Regulatory Networks*
  • Genes, Lethal
  • Genes, Synthetic
  • Humans
  • MRE11 Homologue Protein
  • Molecular Targeted Therapy
  • Mutation
  • RNA, Small Interfering / genetics
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / metabolism

Substances

  • DNA-Binding Proteins
  • MRE11 protein, human
  • RNA, Small Interfering
  • Small Molecule Libraries
  • Flap Endonucleases
  • MRE11 Homologue Protein
  • FEN1 protein, human