A CTG repeat-selective chemical screen identifies microtubule inhibitors as selective modulators of toxic CUG RNA levels.
Proceedings of the National Academy of Sciences of the United States of America. 116(42):20991-21000 [DOI] 10.1073/pnas.1901893116. [PMID] 31570586.
Feb 2019 ACTIVE
Impeding transcription of expanded microsatellite repeats using deactivated Cas9
Funding: NATL INST OF HLTH NIA