- 专利标题： COMPOUNDS FOR IMPROVING MRNA SPLICING
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申请号： EP22178466.3申请日： 2016-01-15
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公开(公告)号： EP4115882A1公开(公告)日： 2023-01-11
- 发明人: SLAUGENHAUPT, Susan, A. , JOHNSON, Graham , PAQUETTE, William, D. , ZHANG, Wei , MARUGAN, Juan
- 申请人： The General Hospital Corporation , The United States of America, as represented by The Secretary, Department of Health and Human Services
- 申请人地址： US Boston, MA 02114 55 Fruit Street; US Bethesda, MD 20852-7660 National Institutes of Health Office of Technology Transfer 6011 Executive Boulevard Suite 325 MSC 7660
- 代理机构： D Young & Co LLP
- 优先权： US201562180380 P 20150616
- 主分类号： A61K31/4196
- IPC分类号： A61K31/4196 ; A61P25/28 ; C07D251/00 ; C07D339/06
摘要：
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.
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