RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
    32.
    发明申请
    RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA) 审中-公开
    RNA干扰介导使用短干扰核酸(siNA)抑制基因表达

    公开(公告)号:US20080161256A1

    公开(公告)日:2008-07-03

    申请号:US11205646

    申请日:2005-08-17

    IPC分类号: A61K31/70 A61P43/00

    CPC分类号: C07H21/02 A61K31/70 A61K48/00

    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. The invention also provides small nucleic acid molecules with reduced or attenuated immunostimulatory properties and methods for designing and synthesizing such small nucleic acid molecules having improved toxicologic properties while retaining RNAi activity.

    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。 本发明还提供具有降低或减弱的免疫刺激性质的小核酸分子以及用于设计和合成具有改善的毒理学特性同时保留RNAi活性的这种小核酸分子的方法。

    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干扰介导使用短干扰核酸(siNA)抑制基因表达

    公开(公告)号:US20070042983A1

    公开(公告)日:2007-02-22

    申请号:US10923536

    申请日:2004-08-20

    IPC分类号: A61K48/00 C07H21/02

    CPC分类号: C12P19/34 A61K48/00 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 interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
    35.
    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 protein tyrosine phosphatase-1B (PTP-1B) gene expression using short interfering nucleic acid (siNA)
    40.
    发明申请
    RNA interference mediated inhibition of protein tyrosine phosphatase-1B (PTP-1B) gene expression using short interfering nucleic acid (siNA) 审中-公开
    RNA干扰介导的使用短干扰核酸(siNA)的蛋白酪氨酸磷酸酶-1B(PTP-1B)基因表达的抑制

    公开(公告)号:US20060025361A1

    公开(公告)日:2006-02-02

    申请号:US11035813

    申请日:2005-01-14

    IPC分类号: A61K48/00 C07H21/04

    摘要: This invention relates to compounds, compositions, and methods useful for modulating protein tyrosine phosphatase-1B (PTP-1B) 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 PTP-1B 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 PTP-1B genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing obesity, insulin resistance, diabetes (eg. type II and type I diabetes) in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of PTP-1B in a cell or tissue, alone or in combination with other treatments or therapies.

    摘要翻译: 本发明涉及使用短干扰核酸(siNA)分子调节蛋白酪氨酸磷酸酶-1B(PTP-1B)基因表达的化合物,组合物和方法。 本发明还涉及使用小核酸分子调节涉及PTP-1B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PTP-1B基因表达的方法。 这样的小核酸分子可用于例如治疗,预防,抑制或减少受试者或生物体内的肥胖,胰岛素抵抗,糖尿病(例如II型和I型糖尿病)和任何其它疾病,性状, 或与细胞或组织中PTP-1B水平相关或将会对其单独或与其它治疗或疗法组合相关的病症。