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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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| Vydáno v: | Nat Chem Biol |
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| Hlavní autoři: | , , , , , , , , , |
| Médium: | Artigo |
| Jazyk: | Inglês |
| Vydáno: |
2019
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| Témata: | |
| On-line přístup: | 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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