BiochemistrybioRxiv
Small molecule targeting r(UGGAA)n disrupts RNA foci and alleviates disease phenotype in Drosophila model
T. Shibata, K. Nagano, M. Ueyama…·K. Nakatani
This paper identifies a small molecule that targets toxic RNA repeats in a Drosophila model of SCA31, reducing RNA foci and alleviating disease symptoms.
DOI 10.1038/s41467-020-20487-4