Inhibition of the expression of huntingtin gene

专利名称：Inhibition of the expression of huntingtin

gene

发明人：Kanazawa Ichiro,Liu Wanzhao,Yu-Lai

Wang,Keiji Wada,Jun Goto,Miho Murata

申请号：US10556711申请日：20040430公开号：US07589189B2公开日：20090915

专利附图：

摘要：It is intended to provide methods for suppressing the huntington geneexpression by using a double-stranded RNA (dsRNA), huntington gene expression

inhibitors to suppress the huntington gene expression, and preventives and/or remediesof Huntington's disease. Targeting against a specific sequence of mRNA at immediatelyupstream of CAG repeats in HD genes of Huntington's disease, the huntington geneexpression 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 21-23bp can be effectively used as the dsRNA homologous to a specific RNA sequence in aregion at immediately upstream of CAG repeats. The dsRNA of this present invention canbe used as a huntington gene expression inhibitor, or a preventive and/or a remedy ofHuntington's disease by administering or introducing into a living body or a living cell inmammals for the prevention and/or treatment of Huntington's disease.

申请人：Kanazawa Ichiro,Liu Wanzhao,Yu-Lai Wang,Keiji Wada,Jun Goto,Miho Murata

地址：Tokyo JP,Tokyo JP,Tokyo JP,Tokyo JP,Tokyo JP,Tokyo JP

国籍：JP,JP,JP,JP,JP,JP

代理机构：Alston & Bird LLP

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