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21.发明授权公开(公告)号：US07749978B2
公开(公告)日：2010-07-06
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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22.发明授权公开(公告)号：US07473525B2
公开(公告)日：2009-01-06
申请号：US10384260
申请日：2003-03-07
申请人： Roland Kreutzer , Stefan Limmer , Hans-Peter Vornlocher , Philipp Hadwiger , Anke Geick , Matthias Ocker , Christoph Herold , Detlef Schuppan
发明人： Roland Kreutzer , Stefan Limmer , Hans-Peter Vornlocher , Philipp Hadwiger , Anke Geick , Matthias Ocker , Christoph Herold , Detlef Schuppan
CPC分类号： C12N15/113 , C12N15/1131 , C12N2310/152 , C12N2310/315 , C12N2310/3183 , C12N2310/33 , C12N2310/351 , C12N2310/3511 , C12N2310/53
摘要： The present invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an anti-apoptotic gene, comprising a complementary RNA strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially identical to at least a part of an apoptotic gene, such as a Bcl 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 an anti-apoptotic gene using the pharmaceutical composition; and methods for inhibiting the expression of an anti-apoptotic gene in a cell.
摘要翻译： 本发明涉及用于抑制抗凋亡基因表达的双链核糖核酸（dsRNA），其包含具有长度小于25个核苷酸的核苷酸序列的互补RNA链，其至少与至少 凋亡基因的一部分，如Bcl基因。 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; 用于使用该药物组合物治疗由抗凋亡基因表达引起的疾病的方法; 以及抑制细胞中抗凋亡基因表达的方法。
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公开(公告)号：US20110092565A1
公开(公告)日：2011-04-21
申请号：US12416140
申请日：2009-03-31
IPC分类号： A61K31/7105 , A61P25/28
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：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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公开(公告)号：US07956177B2
公开(公告)日：2011-06-07
申请号：US12294388
申请日：2007-03-23
IPC分类号： C07H21/04
CPC分类号： C12N15/1137 , C12N15/111 , C12N15/1131 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12N2320/31 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for treating human papilloma virus (HPV) infection. The dsRNA comprises 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 an HPV Target gene selected from among HPV E1, HPV E6 and the human E6AP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HPV infection and the expression of the E6AP gene using the pharmaceutical composition; and methods for inhibiting the expression of the HPV Target genes in a cell.
摘要翻译： 本发明涉及用于治疗人乳头瘤病毒（HPV）感染的双链核糖核酸（dsRNA）。 dsRNA包含具有长度小于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，并且与选自HPV E1，HPV E6中的HPV靶基因的至少一部分基本互补 和人类E6AP基因。 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HPV感染引起的疾病和使用该药物组合物表达E6AP基因的方法; 以及抑制细胞中HPV靶基因表达的方法。
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公开(公告)号：US07812150B2
公开(公告)日：2010-10-12
申请号：US11750553
申请日：2007-05-18
IPC分类号： C07H21/04
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/3515
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 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 an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.
摘要翻译： 本发明涉及用于抑制Aha基因（Aha1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Aha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Aha1表达引起的疾病和使用该药物组合物表达Aha基因的方法; 以及抑制细胞中Aha基因表达的方法。
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公开(公告)号：US20080015161A1
公开(公告)日：2008-01-17
申请号：US11724790
申请日：2007-03-15
CPC分类号： C12N15/1136 , C12N2310/14 , C12N2310/321 , C12N2310/3521
摘要： The present invention concerns methods of treatment using transforming growth factor beta (TGF-beta) modulators. More specifically, the invention concerns methods of treating disorders associated with undesirable TGF-beta signaling, by administering short interfering RNA which down-regulate the expression of TGF-beta, and agents useful therein.
摘要翻译： 本发明涉及使用转化生长因子β（TGF-β）调节剂的治疗方法。 更具体地，本发明涉及通过施用下调TGF-β表达的短干扰RNA以及其中有用的试剂来治疗与不期望的TGF-β信号传导相关的病症的方法。
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公开(公告)号：US20110003882A1
公开(公告)日：2011-01-06
申请号：US12877025
申请日：2010-09-07
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/3515
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of an Aha gene (Aha1 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 an Aha gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by Aha1 expression and the expression of an Aha gene using the pharmaceutical composition; and methods for inhibiting the expression of an Aha gene in a cell.
摘要翻译： 本发明涉及用于抑制Aha基因（Aha1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Aha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Aha1表达引起的疾病和使用该药物组合物表达Aha基因的方法; 以及抑制细胞中Aha基因表达的方法。
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公开(公告)号：US20100249052A1
公开(公告)日：2010-09-30
申请号：US12593202
申请日：2008-03-25
IPC分类号： C12N15/86 , C07H21/02 , A61K31/7088
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3515 , C12Y603/02019 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for treating human papilloma virus (HPV) infection. The dsRNA comprises 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 an HPV Target gene selected from the human E6AP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HPV infection and the expression of the E6AP gene using the pharmaceutical composition; and methods for inhibiting the expression of the HPV Target genes in a cell.
摘要翻译： 本发明涉及用于治疗人乳头瘤病毒（HPV）感染的双链核糖核酸（dsRNA）。 dsRNA包含具有长度小于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，并且其与至少一部分选自人E6AP基因的HPV靶基因互补。 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HPV感染引起的疾病和使用该药物组合物表达E6AP基因的方法; 以及抑制细胞中HPV靶基因表达的方法。
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