Invention Grant
- Patent Title: Compounds for improving mRNA splicing
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Application No.: US15543826Application Date: 2016-01-15
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Publication No.: US10676475B2Publication Date: 2020-06-09
- Inventor: Susan A. Slaugenhaupt , Graham Johnson , William D. Paquette , Wei Zhang , Juan Marugan
- Applicant: The General Hospital Corporation , The United States of America, as Represented by the Secretary, Department of Health and Human Services
- Applicant Address: US MA Boston US MD Bethesda
- Assignee: The General Hospital Corporation,The United States of America, as Represented by the Secretary, Department of Health and Human Services
- Current Assignee: The General Hospital Corporation,The United States of America, as Represented by the Secretary, Department of Health and Human Services
- Current Assignee Address: US MA Boston US MD Bethesda
- Agency: Fish & Richardson P.C.
- International Application: PCT/US2016/013553 WO 20160115
- International Announcement: WO2016/115434 WO 20160721
- Main IPC: C07D473/34
- IPC: C07D473/34 ; C07D405/12 ; C07D417/12 ; C07D495/04 ; C07D513/04 ; C07D473/16 ; C07D471/04 ; C07D487/04 ; A61P25/28

Abstract:
Provided herein are compounds useful for improving mRNA splicing in a cell. Exemplary compounds provided herein are useful for improving mRNA splicing in genes comprising at least one exon ending in the nucleotide sequence CAA. Methods for preparing the compounds and methods of treating diseases of the central nervous system are also provided.
Public/Granted literature
- US20180118748A1 Compounds for Improving mRNA Splicing Public/Granted day:2018-05-03
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