Small molecule targeting r(UGGAA)n disrupts RNA foci and alleviates disease phenotype in Drosophila model
T. Shibata, K. Nagano, M. Ueyama, K. Ninomiya, H. Tetsuro, Y. Nagai, K. Ishikawa, G. Kawai, 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.
bioRxiv · Oct 5, 2020Biochemistry