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    NUCLEIC ACID COMPOUNDS FOR INHIBITING GENE EXPRESSION AND USES THEREOF
    2.
    发明申请
    NUCLEIC ACID COMPOUNDS FOR INHIBITING GENE EXPRESSION AND USES THEREOF 审中-公开
    用于抑制基因表达的核酸化合物及其用途

    公开(公告)号：US20130011922A1

    公开(公告)日：2013-01-10

    申请号：US13327545

    申请日：2011-12-15

    申请人： STEVEN C. QUAY ,  JAMES MCSWIGGEN ,  NARENDRA K. VAISH ,  MOHAMMAD AHMADIAN

    发明人： STEVEN C. QUAY ,  JAMES MCSWIGGEN ,  NARENDRA K. VAISH ,  MOHAMMAD AHMADIAN

    IPC分类号： C07H21/02 ,  C12N5/071

    CPC分类号： C12N15/111 ,  C12N2310/14 ,  C12N2310/533

    摘要： The present disclosure provides RNA molecules, for example, meroduplex ribonucleic acid molecules (mdRNA), and blunt ended double-stranded ribonucleic acid molecules capable of decreasing or silencing expression of a target gene. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a target mRNA. Also provided are methods of decreasing expression of a target gene in a cell or in a subject to treat a disease or condition associated with the target gene.

    摘要翻译： 本公开提供了RNA分子，例如，meroduplex核糖核酸分子（mdRNA）和能够降低或沉默靶基因表达的平端双链核糖核酸分子。 本公开的mdRNA包含至少三条链，其结合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与靶mRNA互补。 还提供减少细胞或受试者中靶基因表达以治疗与靶基因相关的疾病或病症的方法。

    RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
    3.
    发明授权
    RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA) 有权
    使用短干扰核酸（siNA）的RNA干扰介导的呼吸道合胞病毒（RSV）表达的抑制

    公开(公告)号：US08258288B2

    公开(公告)日：2012-09-04

    申请号：US12247971

    申请日：2008-10-08

    申请人： James McSwiggen ,  Leonid Beigelman

    发明人： James McSwiggen ,  Leonid Beigelman

    IPC分类号： C07H21/04

    CPC分类号： C12N15/8218 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  A61K38/00 ,  A61K47/54 ,  A61K47/554 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/1131 ,  C12N15/1138 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/3515 ,  C12N2310/51 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533

    摘要： This invention relates to compounds, compositions, and methods useful for modulating sespiratory syncytial virus (RSV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of RSV gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of RSV genes, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules cocktails thereof. The application also relates to methods of treating diseases and conditions associated with RSV gene expression, such as RSV infection, respiratory failure, bronchiolitis and pneumonia, as well as providing dosing regimens and treatment protocols.

    摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节呼吸道合胞病毒（RSV）基因表达的化合物，组合物和方法。 本发明还涉及用于通过使用小核酸分子调节参与RSV基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的其他基因的表达和活性的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节RSV基因表达的方法，包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒制剂的混合物。 该应用还涉及治疗与RSV基因表达相关的疾病和病症的方法，例如RSV感染，呼吸衰竭，细支气管炎和肺炎，以及提供给药方案和治疗方案。

    RNA INTERFERENCE MEDIATED INHIBITION OF HUMAN IMMUNODEFICIENCY VIRUS (HIV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    4.
    发明申请
    RNA INTERFERENCE MEDIATED INHIBITION OF HUMAN IMMUNODEFICIENCY VIRUS (HIV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
    RNA干扰介导的人类免疫缺陷病毒（HIV）基因表达的抑制使用短暂的干扰核酸（siNA）

    公开(公告)号：US20120041184A1

    公开(公告)日：2012-02-16

    申请号：US13179785

    申请日：2011-07-11

    申请人： Leonid Beigelman ,  James McSwiggen

    发明人： Leonid Beigelman ,  James McSwiggen

    IPC分类号： C07H21/00

    CPC分类号： C07H21/00 ,  C12N15/1132 ,  C12N2310/14

    摘要： This invention relates to compounds, compositions, and methods useful for modulating human immunodeficiency virus (HIV) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of human immunodeficiency virus (HIV) gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of HIV genes. The small nucleic acid molecules are useful in the treatment of HIV infection, AIDS, and/or diseases and conditions related to HIV infection and/or AIDS in a subject or organism.

    摘要翻译： 本发明涉及可用于使用短干扰核酸（siNA）分子调节人免疫缺陷病毒（HIV）基因表达的化合物，组合物和方法。 本发明还涉及可用于通过使用小核酸分子调节涉及人免疫缺陷病毒（HIV）基因表达和/或通过RNA干扰（RNAi）的活性的其它基因的其他基因的表达和活性的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节HIV基因表达的方法。 小核酸分子可用于治疗HIV感染，艾滋病和/或与受试者或生物体中的HIV感染和/或AIDS相关的疾病和病症。

    RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    5.
    发明申请
    RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
    RNA干扰介导的抑制丙糖转化酶底物KEXIN 9（PCSK9）使用短暂的核酸（siNA）的基因表达

    公开(公告)号：US20110313024A1

    公开(公告)日：2011-12-22

    申请号：US13190089

    申请日：2011-07-25

    申请人： LEONID BEIGELMAN ,  JAMES MCSWIGGEN

    发明人： LEONID BEIGELMAN ,  JAMES MCSWIGGEN

    IPC分类号： A61K48/00 ,  A61P3/00 ,  A61P3/10 ,  A61P9/10 ,  A61P9/12 ,  C07H21/02 ,  A61P9/00

    CPC分类号： C12N15/1137 ,  C12N2310/14 ,  C12Y304/21061

    摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression and/or activity. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules.

    摘要翻译： 本发明涉及用于研究，诊断和治疗对Proprotein转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达和/或活性的调节作用的性状，疾病和病症的化合物，组合物和方法。 具体地说，本发明涉及包括小核酸分子如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和双链RNA 能够介导针对Proprotein转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括这种小核酸分子的这种小核酸分子和脂质纳米颗粒（LNP）制剂的混合物。

    RNA interference mediated inhibition of XIAP gene expression using short interfering nucleic acid (siNA)
    8.
    发明授权
    RNA interference mediated inhibition of XIAP gene expression using short interfering nucleic acid (siNA) 有权
    RNA干扰介导的使用短干扰核酸（siNA）的XIAP基因表达的抑制

    公开(公告)号：US07897753B2

    公开(公告)日：2011-03-01

    申请号：US12205113

    申请日：2008-09-05

    申请人： James McSwiggen ,  Leonid Beigelman

    发明人： James McSwiggen ,  Leonid Beigelman

    IPC分类号： C07H21/04 ,  C07H21/02 ,  C12Q1/68

    CPC分类号： C12N15/113 ,  A61K38/00 ,  C12N15/87 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2310/53 ,  C12N2330/30 ,  C12N2310/3521

    摘要： This invention relates to compounds, compositions, and methods useful for modulating XIAP gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of XIAP gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of XIAP genes.

    摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节XIAP基因表达的化合物，组合物和方法。 本发明还涉及可用于通过使用小核酸分子调节参与XIAP基因表达和/或通过RNA干扰（RNAi）的活性的途径的其它基因的表达和活性的化合物，组合物和方法。 特别地，本发明具有小的核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） 用于调节XIAP基因表达的分子和方法。

    RNA interference mediated inhibition of checkpoint kinase-1 (CHK-1) gene expression using short interfering nucleic acid (siNA)
    10.
    发明授权
    RNA interference mediated inhibition of checkpoint kinase-1 (CHK-1) gene expression using short interfering nucleic acid (siNA) 有权
    使用短干扰核酸（siNA）的RNA干扰介导的检查点激酶-1（CHK-1）基因表达的抑制

    公开(公告)号：US07855284B2

    公开(公告)日：2010-12-21

    申请号：US12640489

    申请日：2009-12-17

    申请人： James McSwiggen ,  Leonid Beigelman

    发明人： James McSwiggen ,  Leonid Beigelman

    IPC分类号： C07H21/04

    CPC分类号： C12N15/1137 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/332 ,  C12Y207/11001

    摘要： This invention relates to compounds, compositions, and methods useful for modulating checkpoint kinase (e.g., checkpoint kinase-1 or CHK-1) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of checkpoint kinase gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of checkpoint kinase genes.

    摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节检验点激酶（例如检验点激酶-1或CHK-1）基因表达的化合物，组合物和方法。 本发明还涉及可用于通过使用小核酸分子调节参与检测点激酶基因表达和/或通过RNA干扰（RNAi）的活性的其他基因的表达和活性的化合物，组合物和方法。 特别地，本发明的特征在于小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和短发夹RNA（shRNA） ）分子和用于调节检查点激酶基因表达的方法。