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A new class of mRNA drugs targets poison exons

Nature Biotechnology volume 42, pages 1159–1161 (2024)Cite this article

Companies are targeting poison exons embedded in mRNA transcripts as a strategy to restore wild-type protein abundance and cell fitness in severe pediatric epilepsy and other genetic diseases.

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  1. Dublin, Ireland

    Cormac Sheridan

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  1. Cormac Sheridan
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Sheridan, C. A new class of mRNA drugs targets poison exons. Nat Biotechnol 42, 1159–1161 (2024). https://doi.org/10.1038/s41587-024-02355-4

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