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    RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
    2.
    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

    申请人： David Morrisey ,  James McSwiggen ,  Leonid Beigelman

    发明人： David Morrisey ,  James McSwiggen ,  Leonid Beigelman

    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 respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
    5.
    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)
    6.
    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 XIAP gene expression using short interfering nucleic acid (siNA)
    9.
    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。