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Drug discovery

Kill the messenger where it lives

A mutant repeating DNA sequence produces a toxic RNA molecule that causes the neuromuscular disorder myotonic dystrophy type 1. An ‘antisense’ therapy that targets this RNA in cell nuclei shows promise in mice. See Letter p.111

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Figure 1: How to silence a toxic RNA.

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Correspondence to Peter K. Todd or Henry L. Paulson.

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Todd, P., Paulson, H. Kill the messenger where it lives. Nature 488, 36–37 (2012). https://doi.org/10.1038/488036a

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