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公开(公告)号：US20070161595A1
公开(公告)日：2007-07-12
申请号：US11548890
申请日：2006-10-12
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3517 , C12N2310/3521
摘要： Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells.
摘要翻译： 本发明特征的方面涉及用于抑制α-突触核蛋白（SNCA）基因表达的组合物和方法，例如用于治疗神经变性疾病。 本文特征的针对SNCA基因的抗SNCA试剂可以被修饰以改变有利于神经细胞的分布。
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公开(公告)号：US20050186591A1
公开(公告)日：2005-08-25
申请号：US10991286
申请日：2004-11-17
IPC分类号： A61K20060101 , A61K48/00 , C07H21/02 , C12N15/113 , C12Q1/68
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3517 , C12N2310/3521
摘要： Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells.
摘要翻译： 本发明特征的方面涉及用于抑制α-突触核蛋白（SNCA）基因表达的组合物和方法，例如用于治疗神经变性疾病。 本文特征的针对SNCA基因的抗SNCA试剂可以被修饰以改变有利于神经细胞的分布。
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3.发明申请公开(公告)号：US20080221055A1
公开(公告)日：2008-09-11
申请号：US11944961
申请日：2007-11-26
申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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公开(公告)号：US20110015250A1
公开(公告)日：2011-01-20
申请号：US12754110
申请日：2010-04-05
申请人： David Bumcrot , Pamela Tan , Hans-Peter Vornlocher , Anke Geick
发明人： David Bumcrot , Pamela Tan , Hans-Peter Vornlocher , Anke Geick
IPC分类号： A61K31/713 , C07H21/02 , A61P35/00 , C12N15/63 , C12N5/071
CPC分类号： C12N15/1138 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/314 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/3515 , C12N2330/30 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Eg5 gene (Eg5 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the Eg5 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Eg5 expression and the expression of the Eg5 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Eg5 gene in a cell.
摘要翻译： 本发明涉及用于抑制Eg5基因（Eg5基因）表达的双链核糖核酸（dsRNA），其包含长度小于30个核苷酸的核苷酸序列的反义链，其长度通常为19-25个核苷酸 ，并且其与Eg5基因的至少一部分基本上互补。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 用于治疗由Eg5表达引起的疾病和使用该药物组合物表达Eg5基因的方法; 以及用于抑制细胞中Eg5基因表达的方法。
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5.发明申请公开(公告)号：US20100298405A1
公开(公告)日：2010-11-25
申请号：US12417502
申请日：2009-04-02
申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
IPC分类号： A61K31/713 , C07H21/02 , C12N15/63 , C12N5/10 , A61P25/14
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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公开(公告)号：US08314075B2
公开(公告)日：2012-11-20
申请号：US13252917
申请日：2011-10-04
申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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公开(公告)号：US20120136145A1
公开(公告)日：2012-05-31
申请号：US13165568
申请日：2011-06-21
申请人： David Bumcrot , Pamela Tan , Hans-Peter Vornlocher , Anke Geick
发明人： David Bumcrot , Pamela Tan , Hans-Peter Vornlocher , Anke Geick
IPC分类号： C07H21/02
CPC分类号： C12N15/1138 , C12N15/1137 , C12N2310/111 , C12N2310/14 , C12N2310/314 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/3515 , C12N2330/30 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Eg5 gene (Eg5 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the Eg5 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Eg5 expression and the expression of the Eg5 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Eg5 gene in a cell.
摘要翻译： 本发明涉及用于抑制Eg5基因（Eg5基因）表达的双链核糖核酸（dsRNA），其包含长度小于30个核苷酸的核苷酸序列的反义链，其长度通常为19-25个核苷酸 ，并且其与Eg5基因的至少一部分基本上互补。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 用于治疗由Eg5表达引起的疾病和使用该药物组合物表达Eg5基因的方法; 以及用于抑制细胞中Eg5基因表达的方法。
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8.发明授权公开(公告)号：US08080532B2
公开(公告)日：2011-12-20
申请号：US12417502
申请日：2009-04-02
申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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公开(公告)号：US20070099860A1
公开(公告)日：2007-05-03
申请号：US11588674
申请日：2006-10-27
申请人： Dinah Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
IPC分类号： A61K48/00 , C07H21/04 , C12P21/06 , C07K14/705
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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10.发明授权公开(公告)号：US07320965B2
公开(公告)日：2008-01-22
申请号：US11588674
申请日：2006-10-27
申请人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
发明人： Dinah Wen-Yee Sah , Philipp Hadwiger , Ingo Roehl , Birgit Bramlage , Pamela Tan , Hans-Peter Vornlocher , David Bumcrot
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Huntingtin gene (HD gene), comprising an antisense strand having a nucleotide sequence which is less than 25 nucleotides in length and which is substantially complementary to at least a part of the HD gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of the HD gene, or a mutant form thereof, using the pharmaceutical composition; and methods for inhibiting the expression of the huntingtin gene in a cell.
摘要翻译： 本发明涉及用于抑制亨廷顿蛋白基因（HD基因）表达的双链核糖核酸（dsRNA），其包含长度小于25个核苷酸的核苷酸序列的反义链，并且至少与至少 HD基因的一部分。 本发明还涉及包含dsRNA与药学上可接受的载体的药物组合物; 使用该药物组合物治疗由HD基因的表达引起的疾病或其突变形式的方法; 以及抑制细胞中亨廷顿蛋白基因表达的方法。
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