Cell BiologybioRxiv
A small molecule that binds an RNA repeat expansion stimulates its decay via the exosome complex
A. J. Angelbello, R. I. Benhamou, S. G. Rzuczek…·M. D. Disney
A small molecule binds an RNA repeat expansion, reversing disease defects in DM1 and FECD by either disabling gain-of-function or stimulating exosome-mediated decay.