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Article

Small molecule targeting r(UGGAA)n disrupts RNA foci and alleviates disease phenotype in Drosophila model.

Nature communications - 11 Jan 2021

Shibata Tomonori, Nagano Konami, Ueyama Morio, Ninomiya Kensuke, Hirose Tetsuro, Nagai Yoshitaka, Ishikawa Kinya, Kawai Gota, Nakatani Kazuhiko

Abstract excerpt

Synthetic small molecules modulating RNA structure and function have therapeutic potential for RNA diseases. Here we report our discovery that naphthyridine carbamate dimer (NCD) targets disease-causing r(UGGAA)n repeat RNAs in spinocerebellar ataxia type 31 (SCA31). Structural analysis of the NCD-UGGAA/UGGAA complex by nuclear magnetic resonance (NMR) spectroscopy clarifies the mode of binding that recognizes...

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PMID: 33431896
DOI: 10.1038/s41467-020-20487-4
PMCID: PMC7801683

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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