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1. 发明授权

US07589189B2 Inhibition of the expression of huntingtin gene 失效
标题翻译：抑制亨廷顿蛋白基因的表达
公开(公告)号：US07589189B2
公开(公告)日：2009-09-15
申请号：US10556711
申请日：2004-04-30
申请人： Kanazawa Ichiro , Liu Wanzhao , Yu-Lai Wang , Keiji Wada , Jun Goto , Miho Murata
发明人： Kanazawa Ichiro , Liu Wanzhao , Yu-Lai Wang , Keiji Wada , Jun Goto , Miho Murata
IPC分类号： A01N43/04 , A61K31/70 , C07H21/02 , C07H21/04
CPC分类号： A61K31/7088 , A61K38/00 , C12N15/113 , C12N2310/14
摘要： It is intended to provide methods for suppressing the huntington gene expression by using a double-stranded RNA (dsRNA), huntington gene expression inhibitors to suppress the huntington 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 huntington 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 huntington 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.
摘要翻译：旨在提供通过使用双链RNA（dsRNA），亨廷汀蛋白基因表达抑制剂来抑制亨廷汀蛋白酶基因表达来抑制亨廷顿蛋白基因表达的方法，以及亨廷顿氏病的预防和/或补救措施。在Huntingtin氏病的HD基因中紧邻CAG重复上游的mRNA的特异性序列靶向，通过使用与该序列同源的dsRNA来抑制亨廷顿蛋白基因表达。在本发明中，具有短至约21-23bp的bp的短siRNA（短双链RNA）可以有效地用作在CAG重复的紧邻上游区域中与特定RNA序列同源的dsRNA。本发明的dsRNA可以用作亨廷顿氏基因表达抑制剂，或通过施用或引入哺乳动物的活体或活细胞来预防和/或治疗亨廷顿氏病的亨廷顿氏病的预防和/或补救措施疾病。

2. 发明申请

US20080015158A1 Inhibition of the Expression of Huntingtin Gene 失效
标题翻译：抑制Huntingtin基因的表达
公开(公告)号：US20080015158A1
公开(公告)日：2008-01-17
申请号：US10556711
申请日：2004-04-30
申请人： Kanazawa Ichiro , Liu Wanzhao , Yu-Lai Wang , Keiji Wada , Jun Goto , Miho Murata
发明人： Kanazawa Ichiro , Liu Wanzhao , Yu-Lai Wang , Keiji Wada , Jun Goto , Miho Murata
IPC分类号： A61K31/7105 , A61P25/14 , C07H21/04
CPC分类号： A61K31/7088 , A61K38/00 , C12N15/113 , C12N2310/14
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.
摘要翻译：旨在提供通过使用双链RNA（dsRNA），亨廷汀蛋白酶基因表达抑制剂抑制亨廷顿蛋白基因表达来抑制亨廷顿蛋白基因表达的方法，以及亨廷顿疾病的预防和/或补救措施。针对亨廷顿氏病的HD基因中紧邻CAG重复上游的特异性mRNA序列，通过使用与该序列同源的dsRNA来抑制亨廷顿蛋白基因表达。在本发明中，具有短至约21-23bp的bp的短siRNA（短双链RNA）可以有效地用作在CAG重复的紧邻上游区域中与特定RNA序列同源的dsRNA。本发明的dsRNA可以作为亨廷顿舞蹈病基因表达抑制剂或亨廷顿舞蹈病的预防和/或治疗方法，通过将哺乳动物的活体或活细胞投予或引入，以预防和/或治疗亨廷顿病疾病。

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