The Scripps Research Institute;Disney, Matthew D.;Guan, Lirui;Yang, Wang-yong
发明人:
申请号：
EP14825696.9
公开号：
EP3022219A4
申请日:
2014.07.15
申请国别(地区):
EP
年份:
2017
代理人:
摘要：
The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG)exp sequence which is a cause of myotonic dystrophy type 1 (DM1), or the r(CGG)exp sequence which is a cause of fragile X-associated tremor/ataxia syndrome (FXTAS)； to methods of making the small molecule compounds； and to methods of using the small molecular compounds in the treatment of DM1 or of FXTAS in patients afflicted therewith. The invention further provides a method for identifying an RNA target of a small molecule drug in vivo, using a small molecule drug conjugated to an RNA-reactive crosslinker group and a reporter group, contacting a cell or nucleic acid extract with the small molecule drug conjugate, then separating RNA targets crosslinked to the small molecule drug conjugate by interaction of the affinity group with a complementary affinity group.
