- 专利标题： Compounds and Methods for Modulating Splicing of Pre-MRNA
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申请号： US18363244申请日： 2023-08-01
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公开(公告)号： US20240287513A1公开(公告)日： 2024-08-29
- 发明人: Frank Rigo , Paymaan Jafar-Nejad
- 申请人： Ionis Pharmaceuticals, Inc.
- 申请人地址： US CA Carlsbad
- 专利权人： Ionis Pharmaceuticals, Inc.
- 当前专利权人： Ionis Pharmaceuticals, Inc.
- 当前专利权人地址： US CA Carlsbad
- 主分类号： C12N15/113
- IPC分类号： C12N15/113
摘要：
Provided are compounds, methods, and pharmaceutical compositions for modulating splicing of a pre-mRNA in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a disease or disorder.
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