- 专利标题: Modulation of SMN expression
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申请号: US15565488申请日: 2016-04-11
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公开(公告)号: US10851371B2公开(公告)日: 2020-12-01
- 发明人: Frank Rigo , C. Frank Bennett , Constantin Van Outryve D'Ydewalle , Charlotte J. Sumner
- 申请人: Ionis Pharmaceuticals, Inc. , The Johns Hopkins University
- 申请人地址: US CA Carlsbad US MD Baltimore
- 专利权人: Ionis Pharmaceuticals, Inc.,The Johns Hopkins University
- 当前专利权人: Ionis Pharmaceuticals, Inc.,The Johns Hopkins University
- 当前专利权人地址: US CA Carlsbad US MD Baltimore
- 代理机构: McNeill Baur PLLC
- 国际申请: PCT/US2016/026928 WO 20160411
- 国际公布: WO2016/164896 WO 20161013
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; C07H21/02 ; C12Q1/68
摘要:
Certain embodiments are directed to methods and compounds for inhibiting SMN-NAT, the natural antisense transcript of SMN. Such methods and compounds are useful for increasing expression of SMN in cells and animals.
公开/授权文献
- US20180273943A1 MODULATION OF SMN EXPRESSION 公开/授权日:2018-09-27
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