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公开(公告)号：US20130150570A1
公开(公告)日：2013-06-13
申请号：US13431376
申请日：2012-03-27
申请人： Hans-Peter VORNLOCHER , Ingo ROEHL , Philipp HADWIGER , Tracy Stage ZIMMERMANN , Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Akin AKINC
发明人： Hans-Peter VORNLOCHER , Ingo ROEHL , Philipp HADWIGER , Tracy Stage ZIMMERMANN , Muthiah MANOHARAN , Kallanthottathil G. RAJEEV , Akin AKINC
IPC分类号： C07H21/02
CPC分类号： C07H21/02 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/318 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/53 , C12N2320/51 , C12N2310/3521 , C12N2310/3525 , C12N2310/3527
摘要： This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
摘要翻译： 本发明涉及在细胞和生物流体中具有改善的稳定性的修饰的双链寡核糖核酸（dsRNA），以及制备和鉴定具有改善的稳定性的dsRNA的方法，以及使用dsRNA抑制靶基因的表达或功能。
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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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3.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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4.发明申请公开(公告)号：US20110112176A1
公开(公告)日：2011-05-12
申请号：US12940083
申请日：2010-11-05
申请人： John Frederick Boylan , Birgit Bramlage , Wei He , Markus Hossbach , Ingo Roehl
发明人： John Frederick Boylan , Birgit Bramlage , Wei He , Markus Hossbach , Ingo Roehl
IPC分类号： A61K31/713 , C07H21/02 , C12N15/63 , C12N5/10 , A61P35/00
CPC分类号： C12N15/113 , C12N2310/14 , C12N2310/315 , C12N2310/3515
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a KIF10 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a KIF10 gene using said pharmaceutical composition; and methods for inhibiting the expression of KIF10 in a cell.
摘要翻译： 本发明涉及用于抑制KIF10基因表达的双链核糖核酸（dsRNA）。 本发明还涉及包含dsRNA或核酸分子的药物组合物或其编码与其药学上可接受的载体的载体; 用于使用所述药物组合物治疗由KIF10基因表达引起的疾病的方法; 以及抑制细胞中KIF10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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公开(公告)号：US10711298B2
公开(公告)日：2020-07-14
申请号：US13124411
申请日：2009-10-06
申请人： Ingo Roehl , Markus Schuster , Stephan Seiffert
发明人： Ingo Roehl , Markus Schuster , Stephan Seiffert
IPC分类号： C12Q1/68 , C12Q1/6816 , C07H21/04
摘要： The invention relates to a method for the detection of oligonucleotides using anion exchange high performance liquid chromatography. Fluorescently labelled peptide nucleic acid oligomers, complementary to the oligonucleotide are hybridized to the oligonucleotides. Anion exchange high performance liquid chromatography is then performed and the hybridized moieties detected and quantitated. The invention also relates to a method for the simultaneous detection of both strands of an oligonucleotide in parallel from one sample, and a kit for use in qualitative and quantitative detection of one or two strands of an oligonucleotide.
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公开(公告)号：US20090258934A1
公开(公告)日：2009-10-15
申请号：US12487605
申请日：2009-06-18
申请人： Dinah Sah , Maria Frank-Kamenetsky , Anke Geick , Philipp Hadwiger , Ingo Roehl , Pamela Tan , Hans-Peter Vornlocher
发明人： Dinah Sah , Maria Frank-Kamenetsky , Anke Geick , Philipp Hadwiger , Ingo Roehl , Pamela Tan , Hans-Peter Vornlocher
IPC分类号： A61K31/713 , C07H21/02 , C12N5/10 , C12N15/63 , A61P25/00
CPC分类号： C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Nav1.8 gene (Nav1.8 gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 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 Nav1.8 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Nav1.8 gene gene in a cell.
摘要翻译： 本发明涉及用于抑制Nav1.8基因（Nav1.8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，其基本上 与Nav1.8基因的至少一部分互补。 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; 用于治疗由使用该药物组合物的Nav1.8基因表达引起的疾病的方法; 以及抑制细胞中Nav1.8基因表达的方法。
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公开(公告)号：US07582745B2
公开(公告)日：2009-09-01
申请号：US11593099
申请日：2006-11-03
申请人： Dinah Sah , Maria Frank-Kamenetsky , Anke Geick , Philipp Hadwiger , Ingo Roehl , Pamela Tan , Hans-Peter Vornlocher
发明人： Dinah Sah , Maria Frank-Kamenetsky , Anke Geick , Philipp Hadwiger , Ingo Roehl , Pamela Tan , Hans-Peter Vornlocher
CPC分类号： C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2310/3521
摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the Nav1.8 gene (Nav1.8 gene), comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 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 Nav1.8 gene using the pharmaceutical composition; and methods for inhibiting the expression of the Nav1.8 gene gene in a cell.
摘要翻译： 本发明涉及用于抑制Nav1.8基因（Nav1.8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，其基本上 与Nav1.8基因的至少一部分互补。 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; 用于治疗由使用该药物组合物的Nav1.8基因表达引起的疾病的方法; 以及用于抑制细胞中Nav1.8基因表达的方法。
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9.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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10.发明授权公开(公告)号：US08802773B2
公开(公告)日：2014-08-12
申请号：US13833814
申请日：2013-03-15
申请人： David B Rozema , David L Lewis , Darren H Wakefield , Eric A Kitas , Philipp Hadwiger , Jon A Wolff , Ingo Roehl , Peter Mohr , Torsten Hoffmann , Kerstin Jahn-Hofmann , Hans Martin Mueller , Guenther Ott , Andrei V Blokhin , Jeffrey C Carlson , Jonathan D Benson
发明人： David B Rozema , David L Lewis , Darren H Wakefield , Eric A Kitas , Philipp Hadwiger , Jon A Wolff , Ingo Roehl , Peter Mohr , Torsten Hoffmann , Kerstin Jahn-Hofmann , Hans Martin Mueller , Guenther Ott , Andrei V Blokhin , Jeffrey C Carlson , Jonathan D Benson
CPC分类号： A61K38/00 , A61K31/7088 , A61K47/60 , A61K47/65 , A61K48/00 , C07C69/96 , C07C271/20 , C07H15/04 , C07H15/20 , C07H15/26 , C07K7/02 , C07K2317/77 , C12N15/1138 , C12N15/87 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/351 , C12N2320/30
摘要： The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
摘要翻译： 本发明涉及用于将RNA干扰（RNAi）多核苷酸递送至细胞体内的组合物。 组合物包含用酶切割二肽 - 氨基苄基 - 碳酸酯掩蔽剂可逆地修饰的两亲膜活性多胺。 改性掩盖聚合物的膜活性，而可逆性提供生理反应性。 可逆修饰的多胺（动态多偶联物或DPC）进一步共价连接到RNAi多核苷酸或与靶向RNAi多核苷酸靶向分子缀合物共同施用。
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