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

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 the authors show the mechanism by which a small molecule targets the disease-causing r(UGGAA) n repeat RNAs in spinocerebellar ataxia type 31.

Bibliographic Details
Main Authors: Tomonori Shibata, Konami Nagano, Morio Ueyama, Kensuke Ninomiya, Tetsuro Hirose, Yoshitaka Nagai, Kinya Ishikawa, Gota Kawai, Kazuhiko Nakatani
Format: Article
Language:English
Published: Nature Portfolio 2021-01-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-020-20487-4

Internet

https://doi.org/10.1038/s41467-020-20487-4

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