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mRNA regulation

A patch for a splice

The spliceosome is an immensely complex molecular machine tasked with stitching together coding regions of genes. New work reveals how a small molecule can affect this machinery in a model of spinal muscular atrophy, a disease linked to aberrant splicing.

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Figure 1: Identification of a compound enabling translation of functional SMN.

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Correspondence to Benjamin L Miller.

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Miller, B. A patch for a splice. Nat Chem Biol 11, 454–455 (2015). https://doi.org/10.1038/nchembio.1839

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