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    RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
    11.
    发明申请
    RNA inteference mediated inhibition of hepatitis C virus (HVC) 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）的丙型肝炎病毒（HVC）基因表达的抑制

    公开(公告)号：US20060211642A1

    公开(公告)日：2006-09-21

    申请号：US11311826

    申请日：2005-12-19

    申请人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini

    发明人： James McSwiggen ,  David Morrissey ,  Roberto Guerciolini

    IPC分类号： A61K48/00 ,  A61K9/127

    CPC分类号： C12N15/1131 ,  A61K47/6911 ,  C12N2310/14 ,  C12N2770/24211

    摘要： This invention relates to compounds, compositions, and methods useful for modulating Hepatitis C Virus (HCV) 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 HCV 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 HCV 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 HCV gene expression, such as HCV infection, hepatocellular carcinoma, cirrhosis, liver failure, 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）分子调节丙型肝炎病毒（HCV）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通过RNA干扰（RNAi）的HCV基因表达和/或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HCV基因表达的方法，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HCV基因表达相关的疾病和病症的方法，例如HCV感染，肝细胞癌，肝硬化，肝功能衰竭，以及提供给药方案和治疗方案。

    RNA interference mediated inhibition of STAT3 gene expression using short interfering nucleic acid (siNA)
    13.
    发明申请
    RNA interference mediated inhibition of STAT3 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STAT3基因表达

    公开(公告)号：US20050196781A1

    公开(公告)日：2005-09-08

    申请号：US11014373

    申请日：2004-12-15

    申请人： Howard Robin ,  James McSwiggen

    发明人： Howard Robin ,  James McSwiggen

    IPC分类号： A61K48/00 ,  C07H21/02 ,  C12Q1/68

    CPC分类号： C07H21/02

    摘要： This invention relates to compounds, compositions, and methods useful for modulating STAT3 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 STAT3 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 (mRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of STAT3 genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing cancer, proliferative, and/or inflammatory diseases, disorders, or conditions in a subject or organism, such as psoriasis, eczema, dermatitis, Crohn's disease, and inflammatory bowel disease, and for any other disease, trait, or condition that is related to or will respond to the levels of STAT3 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STAT3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STAT3基因表达和/或通过RNA干扰（RNAi）的活性的其它基因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（mRNA）和短发夹RNA（shRNA） ）分子和用于调节STAT3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中的癌症，增殖性和/或炎性疾病，病症或病症，例如银屑病，湿疹，皮炎，克罗恩病 和炎性肠病，以及与细胞或组织中STAT3水平相关或将会对其单独或与其它治疗或疗法组合反应的任何其它疾病，性状或病症。

    RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
    14.
    发明申请
    RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) 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α-1抗胰蛋白酶（AAT）基因表达

    公开(公告)号：US20050137153A1

    公开(公告)日：2005-06-23

    申请号：US10840731

    申请日：2004-05-06

    申请人： James McSwiggen ,  Howard Robin

    发明人： James McSwiggen ,  Howard Robin

    IPC分类号： A61K38/00 ,  A61K47/48 ,  C12N15/113 ,  C12N15/87 ,  A61K48/00 ,  C07H21/02

    CPC分类号： C12N15/87 ,  A61K38/00 ,  A61K49/0008 ,  C12N15/113 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2310/53 ,  C12N2310/3521

    摘要： The present invention concerns compounds, compositions, and methods for the study, diagnosis, and treatment of diseases and conditions associated with alpha-1 antitrypsin (AAT) allelic variants that respond to the modulation of gene expression and/or activity. The present invention also concerns compounds, compositions, and methods relating to diseases and conditions associated with alpha-1 antitrypsin (AAT) allelic variants that respond to the modulation of expression and/or activity of genes involved in alpha-1 antitrypsin (AAT) gene expression pathways or other cellular processes that mediate the maintenance or development of alpha-1 antitrypsin (AAT) diseases and conditions such as liver disease, lung disease, and any other diseases or conditions that are related to or will respond to the levels of an alpha-1 antitrypsin (AAT) variant protein in a cell or tissue, alone or in combination with other therapies. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (mRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against the expression disease related genes or alleles having alpha-1 antitrypsin (AAT) sequences.

    摘要翻译： 本发明涉及用于研究，诊断和治疗与对基因表达和/或活性的调节作出反应的α-1抗胰蛋白酶（AAT）等位变体相关的疾病和病症的化合物，组合物和方法。 本发明还涉及与涉及α-1抗胰蛋白酶（AAT）基因的基因的表达和/或活性的调节作用的α-1抗胰蛋白酶（AAT）等位变体相关的疾病和病症的化合物，组合物和方法。 介导α-1抗胰蛋白酶（AAT）疾病和病症如肝脏疾病，肺部疾病以及与α水平相关或将会对其反应的任何其他疾病或病症的介导维持或发展的表达途径或其他细胞过程 -1抗胰蛋白酶（AAT）变体蛋白在细胞或组织中，单独或与其他疗法组合。 具体地，本发明涉及小核酸分子，例如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（mRNA）和短发夹RNA（shRNA） 能够介导RNA干扰（RNAi）对具有α-1抗胰蛋白酶（AAT）序列的疾病相关基因或等位基因的表达的分子。

    RNA interference mediated of inhibition of influenza virus gene expression using short interfering nucleic acid (siNA)
    15.
    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

    申请人： Vasant Jadhav ,  Howard Robin ,  James McSwiggen

    发明人： Vasant Jadhav ,  Howard Robin ,  James McSwiggen

    IPC分类号： A61K48/00 ,  C07H21/02

    CPC分类号： C12N15/1131 ,  C12N15/115 ,  C12N2310/14

    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。

    RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
    17.
    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)
    18.
    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。