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

Taladriz-Sender, Andrea and Campbell, Emma and Burley, Glenn A. (2019) Splice-switching small molecules : a new therapeutic approach to modulate gene expression. [Review]

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    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.