The Scripps Research Institute

Guan, Lirui

Disney, Matthew D.

Yang, Wang-yong

Lirui Guan

Matthew D Disney

Wang-Yong Yang

The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG)sequence which is a cause of myotonic dystrophy type 1 (DM1), or the r(CGG)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.

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Rockville, MD, United States of America

Lirui Guan