Structural basis of a small molecule targeting RNA for a specific splicing correction

Splicing modifiers promoting SMN2 exon 7 inclusion have the potential to treat spinal muscular atrophy, the leading genetic cause of infantile death. The small molecules are SMN2 exon 7 selective and act during the early stages of spliceosome assembly. Here, we show at atomic resolution how the drug...

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Pubblicato in:Nat Chem Biol
Autori principali: Campagne, Sébastien, Boigner, Sarah, Rüdisser, Simon, Moursy, Ahmed, Gillioz, Laurent, Knörlein, Anna, Hall, Jonathan, Ratni, Hasane, Cléry, Antoine, Allain, Frédéric H-T.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2019
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7617061/
https://ncbi.nlm.nih.gov/pubmed/31636429
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41589-019-0384-5
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