A CTG repeat-selective chemical screen identifies microtubule inhibitors as selective modulators of toxic CUG RNA levels [Biochemistry]

A CTG repeat expansion in the DMPK gene is the causative mutation of myotonic dystrophy type 1 (DM1). Transcription of the expanded CTG repeat produces toxic gain-of-function CUG RNA, leading to disease symptoms. A screening platform that targets production or stability of the toxic CUG RNA in a selective manner...

Source: Proceedings of the National Academy of Sciences - October 14, 2019 Category: Science Authors: Kaalak Reddy, Jana R. Jenquin, Ona L. McConnell, John D. Cleary, Jared I. Richardson, Belinda S. Pinto, Maja C. Haerle, Elizabeth Delgado, Lori Planco, Masayuki Nakamori, Eric T. Wang, J. Andrew Berglund Tags: PNAS Plus Source Type: research