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公开(公告)号:US09006198B2
公开(公告)日:2015-04-14
申请号:US13577622
申请日:2011-02-08
申请人: C. Frank Bennett , Michael Hayden , Susan M. Freier , Sarah Greenlee , Jeffrey Carroll , Simon Warby , Eric E. Swayze
发明人: C. Frank Bennett , Michael Hayden , Susan M. Freier , Sarah Greenlee , Jeffrey Carroll , Simon Warby , Eric E. Swayze
IPC分类号: C12N15/11 , C12Q1/68 , C12N15/113
CPC分类号: C12Q1/6883 , C12N15/113 , C12N2310/11 , C12N2320/34 , C12Q1/6811 , C12Q1/6897 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , C12Q2525/207 , C12Q2535/131 , C12Q2539/107 , C12Q2521/327 , C12Q2525/121 , C12Q2525/185
摘要: Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).
摘要翻译: 本文公开了用于选择性降低含有单核苷酸多态性(SNP)的亨廷顿氏基因的等位变体的表达的反义化合物和方法。 这些方法,化合物和组合物可用于治疗,预防或改善亨廷顿病(HD)。
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2.
公开(公告)号:US08679750B2
公开(公告)日:2014-03-25
申请号:US12991883
申请日:2009-05-08
申请人: Michael Hayden , Jeffrey Carroll , Simon Warby
发明人: Michael Hayden , Jeffrey Carroll , Simon Warby
CPC分类号: C12Q1/6883 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/34 , G01N33/5023 , G01N2800/2835
摘要: Methods and compositions for reducing expression of a mutant huntingtin (mHTT) protein in a cell are provided. Such methods include contacting the cell with an effective amount of a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding the mHTT.
摘要翻译: 提供了用于降低细胞中突变型亨廷丁蛋白(mHTT)蛋白表达的方法和组合物。 这样的方法包括使细胞与有效量的靶向编码mHTT的RNA的差异多态性的核酸沉默剂接触。
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公开(公告)号:US20130046008A1
公开(公告)日:2013-02-21
申请号:US13577622
申请日:2011-02-08
申请人: C. Frank Bennett , Michael Hayden , Susan M. Freier , Sarah Greenlee , Jeffrey Carroll , Simon Warby , Eric E. Swayze
发明人: C. Frank Bennett , Michael Hayden , Susan M. Freier , Sarah Greenlee , Jeffrey Carroll , Simon Warby , Eric E. Swayze
IPC分类号: A61K31/7088 , A61P25/28 , A61K31/7115 , A61K31/7125 , C12N5/071 , A61K31/712
CPC分类号: C12Q1/6883 , C12N15/113 , C12N2310/11 , C12N2320/34 , C12Q1/6811 , C12Q1/6897 , C12Q2600/136 , C12Q2600/156 , C12Q2600/158 , C12Q2525/207 , C12Q2535/131 , C12Q2539/107 , C12Q2521/327 , C12Q2525/121 , C12Q2525/185
摘要: Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate Huntington's Disease (HD).
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公开(公告)号:US20110213010A1
公开(公告)日:2011-09-01
申请号:US12991883
申请日:2009-05-08
申请人: Michael Hayden , Jeffrey Carroll , Simon Warby
发明人: Michael Hayden , Jeffrey Carroll , Simon Warby
IPC分类号: A61K31/7088 , C12N5/071 , C12Q1/68 , A61P25/28
CPC分类号: C12Q1/6883 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/14 , C12N2320/34 , G01N33/5023 , G01N2800/2835
摘要: Methods and compositions for reducing expression of a mutant huntingtin (mHTT) protein in a cell are provided. Such methods include contacting the cell with an effective amount of a nucleic acid silencing agent targeting a differentiating polymorphism in RNA encoding the mHTT.
摘要翻译: 提供了用于降低细胞中突变型亨廷丁蛋白(mHTT)蛋白表达的方法和组合物。 这样的方法包括使细胞与有效量的靶向编码mHTT的RNA的差异多态性的核酸沉默剂接触。
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