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, S. Choudhary, Z. Tang, J. L. Chen, M. Roy, K. W. Wang, I. Yildirim, A. S. Jun, C. A. Thornton, 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.
bioRxiv · May 11, 2020Cell Biology