Invention Grant
- Patent Title: Specific targeting of RNA expanded repeat sequences
- Patent Title (中): RNA扩增重复序列的特异性靶向
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Application No.: US14904999Application Date: 2014-07-15
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Publication No.: US09586944B2Publication Date: 2017-03-07
- Inventor: Matthew D. Disney , Lirui Guan , Wang-Yong Yang
- Applicant: THE SCRIPPS RESEARCH INSTITUTE
- Applicant Address: US CA La Jolla
- Assignee: The Scripps Research Institute
- Current Assignee: The Scripps Research Institute
- Current Assignee Address: US CA La Jolla
- Agent Geoffrey K. Cooper; Thomas Fitting
- International Application: PCT/US2014/046626 WO 20140715
- International Announcement: WO2015/009678 WO 20150122
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C07D403/14 ; C07D495/04 ; C07D519/00

Abstract:
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.
Public/Granted literature
- US20160257669A1 SPECIFIC TARGETING OF RNA EXPANDED REPEAT SEQUENCES Public/Granted day:2016-09-08
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