METHODS FOR TREATING AND PREVENTING APOPTOSIS-RELATED DISEASES USING RNA INTERFERING AGENTS
    3.
    发明申请
    METHODS FOR TREATING AND PREVENTING APOPTOSIS-RELATED DISEASES USING RNA INTERFERING AGENTS 审中-公开
    使用RNA干扰剂治疗和预防病毒性相关疾病的方法

    公开(公告)号:WO2005013886A2

    公开(公告)日:2005-02-17

    申请号:PCT/US2003/034686

    申请日:2003-10-30

    IPC: A61K

    Abstract: The present invention is based, at least in part, on the discovery of compositions and methods useful in the modulation, e.g ., inhibition, of gene expression or protein activity. In particular, the present invention is based on novel RNA interfering agents, e.g ., siRNA molecules which target apoptosis-related genes or proinflammatory cytokines, and result in reduction, e.g ., prolonged reduction, of apoptosis-related gene expression or proinflammatory cytokine expression in cells. Inhibition of apoptosis­related gene expression or protein activity or proinflammatory cytokine expression or protein activity, e.g ., by the siRNAs of the invention, inhibits apoptosis-mediated diseases or disorders and proinflammatory cytokine mediated diseases or disorders, including, for example, transplant rejection, hepatitis, liver injury, sepsis, and cancer.

    Abstract translation: 本发明至少部分地基于发现可用于调节,例如抑制基因表达或蛋白质活性的组合物和方法。 特别地,本发明基于新的RNA干扰剂,例如靶向凋亡相关基因或促炎细胞因子的siRNA分子,并导致细胞凋亡相关基因表达或促炎细胞因子表达的减少,例如延长的减少 。 抑制凋亡相关基因表达或蛋白质活性或促炎细胞因子表达或蛋白质活性,例如本发明的siRNA抑制凋亡介导的疾病或病症和促炎细胞因子介导的疾病或病症,包括例如移植排斥,肝炎, 肝损伤,败血症和癌症。

    INHIBITION OF GENE EXPRESSION USING RNA INTERFERING AGENTS
    6.
    发明申请
    INHIBITION OF GENE EXPRESSION USING RNA INTERFERING AGENTS 审中-公开
    用RNA干扰因子抑制基因表达

    公开(公告)号:WO2004039957A3

    公开(公告)日:2004-09-30

    申请号:PCT/US0334424

    申请日:2003-10-29

    Abstract: The present invention is based, at least in part, on the discovery of compositions and methods for the treatment and prevention of infectious diseases or disorders, e.g., HIV infection, AIDS, and AIDS-related diseases. In particular, the present invention pertains to methods of modulating cellular gene expression or protein activity, e.g., CCR5, gene expression or protein activity and/or gene expression or protein activity of a gene or sequence of an infectious agent, in order to treat or prevent infectious diseases or disorders, HIV infection, AIDS, or an AIDS-related disease or disorder. In one embodiment, the combination of an RNA interfering agent targeting a cellular gene in combination with an RNA interfering agent targeting a gene or sequence of an infectious agent results in prolonged prevention of infection by an infectious agent. The present invention is based on the identification of novel RNA interference agents, e.g., siRNA molecules, which target cellular genes, e.g., chemokine receptors, e.g., the CCR5 gene, and result in inhibition of target gene expression on target gene expressing cells, thereby inhibiting entry of infectious agents, e,g., HIV infection into target cells, prevention infection, and/or suppressing replication in established infection.

    Abstract translation: 本发明至少部分基于发现用于治疗和预防感染性疾病或病症例如HIV感染,AIDS和AIDS相关疾病的组合物和方法。 具体而言,本发明涉及调节细胞基因表达或蛋白质活性(例如CCR5),基因表达或蛋白质活性和/或基因或感染因子序列的基因表达或蛋白质活性的方法,以治疗或 预防感染性疾病或病症,HIV感染,艾滋病或与AIDS相关的疾病或病症。 在一个实施方案中,靶向细胞基因的RNA干扰剂与靶向感染剂基因或序列的RNA干扰剂的组合导致延长的对传染因子感染的预防。 本发明基于鉴定靶向细胞基因(例如趋化因子受体,例如CCR5基因)的新型RNA干扰试剂例如siRNA分子,并且导致抑制靶基因表达细胞上的靶基因表达,由此 抑制感染因子例如HIV感染进入靶细胞,预防感染和/或抑制已建立的感染中的复制。

    INHIBITION OF GENE EXPRESSION USING RNA INTERFERING AGENTS
    7.
    发明申请
    INHIBITION OF GENE EXPRESSION USING RNA INTERFERING AGENTS 审中-公开
    使用RNA干扰剂抑制基因表达

    公开(公告)号:WO2004039957A2

    公开(公告)日:2004-05-13

    申请号:PCT/US2003/034424

    申请日:2003-10-29

    IPC: C12N

    Abstract: The present invention is based, at least in part, on the discovery of compositions and methods for the treatment and prevention of infectious diseases or disorders, e.g., HIV infection, AIDS, and AIDS-related diseases. In particular, the present invention pertains to methods of modulating cellular gene expression or protein activity, e.g., CCR5, gene expression or protein activity and/or gene expression or protein activity of a gene or sequence of an infectious agent, in order to treat or prevent infectious diseases or disorders, HIV infection, AIDS, or an AIDS-related disease or disorder. In one embodiment, the combination of an RNA interfering agent targeting a cellular gene in combination with an RNA interfering agent targeting a gene or sequence of an infectious agent results in prolonged prevention of infection by an infectious agent. The present invention is based on the identification of novel RNA interference agents, e.g., siRNA molecules, which target cellular genes, e.g., chemokine receptors, e.g., the CCR5 gene, and result in inhibition of target gene expression on target gene expressing cells, thereby inhibiting entry of infectious agents, e,g., HIV infection into target cells, prevention infection, and/or suppressing replication in established infection.

    Abstract translation: 本发明至少部分地基于发现用于治疗和预防感染性疾病或病症的组合物和方法,例如HIV感染,AIDS和AIDS相关疾病。 特别地,本发明涉及调节细胞基因表达或蛋白质活性的方法,例如CCR5,基因表达或蛋白质活性和/或感染因子的基因或序列的基因表达或蛋白质活性,以便治疗或 预防传染病或疾病,艾滋病毒感染,艾滋病或艾滋病相关疾病或病症。 在一个实施方案中,靶向细胞基因的RNA干扰剂与靶向感染因子的基因或序列的RNA干扰剂的组合导致长期预防感染因子的感染。 本发明基于鉴定靶向细胞基因(例如趋化因子受体,例如CCR5基因)的新型RNA干扰剂,例如siRNA分子,并导致靶基因表达细胞的靶基因表达的抑制,从而 抑制感染因子的侵入,例如艾滋病毒感染进入靶细胞,预防感染和/或抑制已知感染中的复制。

    METHOD TO TREAT FLAVIVIRUS INFECTION WITH SIRNA
    9.
    发明申请
    METHOD TO TREAT FLAVIVIRUS INFECTION WITH SIRNA 审中-公开
    用SIRNA治疗黄病毒感染的方法

    公开(公告)号:WO2007044468A3

    公开(公告)日:2007-09-13

    申请号:PCT/US2006038980

    申请日:2006-10-05

    Abstract: The present invention is directed to methods of treating flavivirus mediated diseases using siRNAs. The invention is based upon our findings in a mouse model that siRNAs directed against sequences conserved among multiple flaviviruses prevents and treats flavivirus infections. Accordingly, the present invention provides an isolated siRNA comprising a sense RNA and an antisense RNA strand or a single strand. The sense and the antisense RNA strands, or the single RNA strand, form an RNA duplex, and wherein the RNA strand comprises a nucleotide sequence identical to a target sequence of about 15 to about 30 contiguous nucleotides in flavivirus mRNA or mutant or variant thereof.

    Abstract translation: 本发明涉及使用siRNA治疗黄病毒介导的疾病的方法。 本发明基于我们在小鼠模型中的发现,即针对多种黄病毒中保守序列的siRNA预防和治疗黄病毒感染。 因此,本发明提供了包含有义RNA和反义RNA链或单链的分离的siRNA。 正义和反义RNA链或单个RNA链形成RNA双链体,并且其中RNA链包含与黄病毒mRNA或其突变体或变体中的约15至约30个连续核苷酸的靶序列相同的核苷酸序列。

    METHODS FOR TREATING AND PREVENTING APOPTOSIS-RELATED DISEASES USING RNA INTERFERING AGENTS
    10.
    发明申请
    METHODS FOR TREATING AND PREVENTING APOPTOSIS-RELATED DISEASES USING RNA INTERFERING AGENTS 审中-公开
    使用RNA干扰剂治疗和预防病毒性相关疾病的方法

    公开(公告)号:WO2005013886A3

    公开(公告)日:2006-03-23

    申请号:PCT/US0334686

    申请日:2003-10-30

    Abstract: The present invention is based, at least in part, on the discovery of compositions and methods useful in the modulation, e.g., inhibition, of gene expression or protein activity. In particular, the present invention is based on novel RNA interfering agents, e.g., siRNA molecules which target apoptosis-related genes or proinflammatory cytokines, and result in reduction, e.g., prolonged reduction, of apoptosis-related gene expression or proinflammatory cytokine expression in cells. Inhibition of apoptosis­related gene expression or protein activity or proinflammatory cytokine expression or protein activity, e.g., by the siRNAs of the invention, inhibits apoptosis-mediated diseases or disorders and proinflammatory cytokine mediated diseases or disorders, including, for example, transplant rejection, hepatitis, liver injury, sepsis, and cancer.

    Abstract translation: 本发明至少部分地基于发现可用于调节,例如抑制基因表达或蛋白质活性的组合物和方法。 特别地,本发明基于新的RNA干扰剂,例如靶向凋亡相关基因或促炎细胞因子的siRNA分子,并导致细胞凋亡相关基因表达或促炎细胞因子表达的减少,例如延长的减少 。 抑制凋亡相关基因表达或蛋白质活性或促炎细胞因子表达或蛋白质活性,例如本发明的siRNA抑制凋亡介导的疾病或病症和促炎细胞因子介导的疾病或病症,包括例如移植排斥,肝炎, 肝损伤,败血症和癌症。

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