Splicing modulators: on the way from nature to clinic

J Antibiot (Tokyo). 2021 Oct;74(10):603-616. doi: 10.1038/s41429-021-00450-1. Epub 2021 Aug 3.


Over the course of more than two decades, natural products isolated from various microorganisms and plants have built the foundation for chemical biology research into the mechanism of pre-mRNA splicing. Hand in hand with advances in scientific methodology small molecule splicing modulators have become powerful tools for investigating, not just the splicing mechanism, but also the cellular effect of altered mRNA processing. Based on thorough structure-activity studies, synthetic analogues have moved on from scientific tool compounds to experimental drugs. With current advances in drug discovery methodology and new means of attacking targets previously thought undruggable, we can expect further advances in both research and therapeutics based on small molecule splicing modulators.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Alternative Splicing
  • Animals
  • Antineoplastic Agents / chemistry*
  • Antineoplastic Agents / metabolism
  • Antineoplastic Agents / pharmacology*
  • Biological Products / chemistry*
  • Biological Products / metabolism
  • Biological Products / pharmacology*
  • Humans


  • Antineoplastic Agents
  • Biological Products