Targeting splicing abnormalities in cancer

Curr Opin Genet Dev. 2018 Feb;48:67-74. doi: 10.1016/j.gde.2017.10.010. Epub 2017 Nov 12.


Recently splicing has been recognized as a key pathway in cancer. Although aberrant splicing has been shown to be a consequence of mutations or the abnormal expression of splicing factors (trans-effect changes) or mutations in the splicing sequences (cis-effect mutations), the connections between aberrant splicing and cancer initiation or progression are still not well understood. Here we review the mutational landscape of splicing factors in cancer and associated splicing consequences, along with the most important examples of the therapeutic approaches targeting the spliceosome currently being investigated in oncology.

Publication types

  • Review

MeSH terms

  • Animals
  • Humans
  • Mutation
  • Neoplasms / drug therapy*
  • Neoplasms / genetics*
  • Oligonucleotides / therapeutic use*
  • RNA Splicing / drug effects*
  • RNA Splicing Factors / genetics
  • RNA Splicing Factors / metabolism
  • Spliceosomes / drug effects
  • Spliceosomes / metabolism


  • Oligonucleotides
  • RNA Splicing Factors