发明名称 |
INHIBITION OF THE EXPRESSION OF HUNTINGTIN GENE |
摘要 |
It is intended to provide methods for suppressing the huntingtin gene expression by using a double-stranded RNA (dsRNA), huntingtin gene expression inhibitors to suppress the huntingtin gene expression, and preventives and/or remedies of Huntington's disease. Targeting against a specific sequence of mRNA at immediately upstream of CAG repeats in HD genes of Huntington's disease, the huntingtin gene expression is suppressed by using a dsRNA homologous to the sequence. In this invention, a short siRNA (short double-stranded RNA) having bp as short as around 21-23 bp can be effectively used as the dsRNA homologous to a specific RNA sequence in a region at immediately upstream of CAG repeats. The dsRNA of this present invention can be used as a huntingtin gene expression inhibitor, or a preventive and/or a remedy of Huntington's disease by administering or introducing into a living body or a living cell in mammals for the prevention and/or treatment of Huntington's disease.
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申请公布号 |
CA2526893(C) |
申请公布日期 |
2010.10.26 |
申请号 |
CA20042526893 |
申请日期 |
2004.04.30 |
申请人 |
JAPAN SCIENCE AND TECHNOLOGY AGENCY |
发明人 |
KANAZAWA, ICHIRO;LIU, WANZHAO;WANG, YU-LAI;WADA, KEIJI;GOTO, JUN;MURATA, MIHO |
分类号 |
A61K31/7088;A61K38/00;A61K48/00;A61P25/14;C12N15/113 |
主分类号 |
A61K31/7088 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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