Splice-switching small molecules: a new therapeutic approach to modulate gene expression

Research output: Contribution to journalBook/Film/Article review

3 Citations (Scopus)

Abstract

Manipulating alternative RNA splicing events with small molecules is emerging as a viable mechanism for the development of therapeutics. A salient challenge in the field is understanding the molecular determinants defining the selectivity of splice-switching events and their mechanisms of action. In this review, the current state-of-the-art in splice-switching small molecules is described. Three examples of splice-switching small molecules are presented, and the differences in their modes of action compared.
Original languageEnglish
Pages (from-to)134-142
Number of pages9
JournalMethods
Volume167
Early online date14 Jun 2019
DOIs
Publication statusPublished - 1 Sep 2019

Keywords

  • RNA splicing
  • small molecules
  • splice switching

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