Targeting splicing factors for cancer therapy

Ariel Bashari, Zahava Siegfried, Rotem Karni

Research output: Contribution to journalArticlepeer-review


Alternative splicing (AS) of mRNAs is an essential regulatory mechanism in eukaryotic gene expression. AS misregulation, caused by either dysregulation or mutation of splicing factors, has been shown to be involved in cancer development and progression, making splicing factors suitable targets for cancer therapy. In recent years, various types of pharmacological modulators, such as small molecules and oligonucleotides, targeting distinct components of the splicing machinery, have been under development to treat multiple disorders. Although these approaches have promise, targeting the core spliceosome components disrupts the early stages of spliceosome assembly and can lead to nonspecific and toxic effects. New research directions have been focused on targeting specific splicing factors for a more precise effect. In this Perspective, we will highlight several approaches for targeting splicing factors and their functions and suggest ways to improve their specificity.

Original languageAmerican English
Pages (from-to)506-515
Number of pages10
Issue number4
StatePublished - Apr 2023


  • cancer therapy
  • decoy oligonucleotides
  • small molecules
  • splicing
  • splicing factor

All Science Journal Classification (ASJC) codes

  • Molecular Biology


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