M. Ueyama

Reconstitution of C9orf72 (GGGGCC) repeat-associated non-AUG translation with purified components.

This study reconstitutes G4C2 repeat-associated non-AUG translation using purified human factors and identifies key features of the process.

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

This paper identifies a small molecule that targets toxic RNA repeats in a Drosophila model of SCA31, reducing RNA foci and alleviating disease symptoms.