RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
    31.
    发明申请
    RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA) 审中-公开
    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:US20060287267A1

    公开(公告)日:2006-12-21

    申请号:US11395833

    申请日:2006-03-31

    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 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) )分子和方法用于调节RSV基因的表达,包括这种小核酸分子的混合物和这种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 HEPATITIS C VIRUS (HCV) EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

    公开(公告)号:US20090306184A1

    公开(公告)日:2009-12-10

    申请号:US12418477

    申请日:2009-04-03

    IPC分类号: A61K31/7105 C07H21/02

    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 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. 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 gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.

    RNA interference mediated inhibition of 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) gene expression using short interfering nucleic acid siNA

    公开(公告)号:US20070049543A1

    公开(公告)日:2007-03-01

    申请号:US11455205

    申请日:2006-06-16

    IPC分类号: A61K48/00 C07H21/02

    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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. 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 11 beta-hydroxysteroid dehydrogenase-1 (11 beta-HSD-1) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate 11 beta-HSD-1 gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce metabolic diseases traits and conditions, including but not limited to diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity, hypercholesterolemia, cardiovascular disease and/or other disease states, conditions, or traits associated with 11 beta-HSD-1 gene expression or activity in a subject or organism.

    RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    34.
    发明申请
    RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
    RNA干扰介导的基因表达抑制使用短暂的干扰核酸(siNA)

    公开(公告)号:US20100145038A1

    公开(公告)日:2010-06-10

    申请号:US12615146

    申请日:2009-11-09

    IPC分类号: C07H21/02

    CPC分类号: C07H21/02

    摘要: This invention relates to compounds, compositions, and methods useful for modulating gene expression using short interfering nucleic acid (siNA) 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 genes, such as expressed pseudogenes associated with the maintenance or development of diseases, disorders, traits, and conditions 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)分子调节基因表达的化合物,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) )分子和用于调节基因表达的方法,例如与受试者或生物中的疾病,病症,性状和病症的维持或发展相关的表达的假基因。

    RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)

    公开(公告)号:US20080207542A1

    公开(公告)日:2008-08-28

    申请号:US11510872

    申请日:2006-08-25

    IPC分类号: A61K31/713

    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 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. 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 gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits and conditions that are associated with gene expression or activity in a subject or organism.

    RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)
    38.
    发明授权
    RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA) 有权
    使用短干扰核酸(siNA)的RNA干扰介导的丙型肝炎病毒(HCV)表达的抑制

    公开(公告)号:US07935812B2

    公开(公告)日:2011-05-03

    申请号:US12418477

    申请日:2009-04-03

    IPC分类号: C07H21/04 C07H21/02 A61K48/00

    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 hepatitis C virus (HCV) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in hepatitis C virus (HCV) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules capable of mediating or that mediate RNA interference (RNAi) against hepatitis C virus (HCV) 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,诊断和治疗对丙型肝炎病毒(HCV)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。 本发明还涉及与涉及丙型肝炎病毒(HCV)基因表达途径或介导的其他细胞过程的基因的表达和/或活性的调节作用的性状,疾病和病症有关的化合物,组合物和方法 维持或发展这些性状,疾病和状况。 具体地,本发明涉及能够介导或介导针对丙型肝炎病毒(HCV)基因表达的RNA干扰(RNAi)的双链核酸分子,包括这种小核酸分子的混合物和这种小的核酸分子的脂质纳米颗粒(LNP)制剂 核酸分子。

    RNA interference mediated of inhibition of influenza virus gene expression using short interfering nucleic acid (siNA)
    39.
    发明申请
    RNA interference mediated of inhibition of influenza virus gene expression using short interfering nucleic acid (siNA) 审中-公开
    使用短干扰核酸(siNA)介导的流感病毒基因表达抑制的RNA干扰

    公开(公告)号:US20070099858A1

    公开(公告)日:2007-05-03

    申请号:US11242567

    申请日:2005-10-03

    IPC分类号: A61K48/00 C07H21/02

    摘要: This invention relates to compounds, compositions, and methods useful for modulating influenza virus gene expression using short interfering nucleic acid (siNA) 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 influenza virus genes, such as viral and host genes associated with the maintenance or development of influenza virus infection and related diseases and conditions 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)分子调节流感病毒基因表达的化合物,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) )分子和用于调节流感病毒基因表达的方法,例如与维持或发展流感病毒感染以及受试者或生物体中的相关疾病和病症相关的病毒和宿主基因。