- 专利标题: Selective Reduction of Allelic Variants
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申请号: US17577832申请日: 2022-01-18
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公开(公告)号: US20220403386A1公开(公告)日: 2022-12-22
- 发明人: C. Frank Bennett , Susan M. Freier , Sarah Greenlee , Eric E. Swayze
- 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:
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).
公开/授权文献
- US12110491B2 Selective reduction of allelic variants 公开/授权日:2024-10-08
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