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mRNA splicing drugs

Splice-modifying drug mechanisms

A new study combines massively parallel assays, transcriptomics and biophysical modeling to provide a framework for analyzing the effects of compounds that modulate pre-mRNA splicing. The results lend important insights into the mechanisms of drug action and facilitate the design of splicing therapies.

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Fig. 1: Therapeutic mechanism of splicing modulation for SMA and molecular basis for the specificities of risdiplam and branaplam.

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Correspondence to Juan Valcárcel.

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Competing interests

J.V. is a member of the scientific advisory boards of Remix Therapeutics, Stoke Therapeutics and IntronX.

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Herrero-Vicente, J., Black, D.L. & Valcárcel, J. Splice-modifying drug mechanisms. Nat Chem Biol 20, 1103–1105 (2024). https://doi.org/10.1038/s41589-024-01678-2

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