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

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...

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Detalles Bibliográficos
Autores principales: Shibata, Tomonori, Nagano, Konami, Ueyama, Morio, Ninomiya, Kensuke, Hirose, Tetsuro, Nagai, Yoshitaka, Ishikawa, Kinya, Kawai, Gota, Nakatani, Kazuhiko
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7801683/
https://www.ncbi.nlm.nih.gov/pubmed/33431896
http://dx.doi.org/10.1038/s41467-020-20487-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7801683/
https://www.ncbi.nlm.nih.gov/pubmed/33431896
http://dx.doi.org/10.1038/s41467-020-20487-4

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