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    RNA INTERFERENCE MEDIATED INHIBITION OF MATRIX METALLOPROTEINASE 13 (MMP13) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    81.
    发明申请
    RNA INTERFERENCE MEDIATED INHIBITION OF MATRIX METALLOPROTEINASE 13 (MMP13) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 有权
    RNA干扰介导的基质金属蛋白酶13（MMP13）抑制基因表达使用短暂的核酸（siNA）

    公开(公告)号：US20090099121A1

    公开(公告)日：2009-04-16

    申请号：US12204612

    申请日：2008-09-04

    申请人： James McSwiggen ,  Leonid Beigelman

    发明人： James McSwiggen ,  Leonid Beigelman

    IPC分类号： A61K31/712 ,  C07H1/02

    CPC分类号： C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  C07H21/00 ,  C07H21/04 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3521

    摘要： This invention relates to compounds, compositions, and methods useful for modulating matrix metalloproteinase (e.g., MMP13) 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 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 MMP13 genes.

    摘要翻译： 本发明涉及可用于使用短干扰核酸（siNA）分子调节基质金属蛋白酶（例如MMP13）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通过RNA干扰（RNAi）的基因表达和/或活性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MMP13基因表达的方法。

    RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR CELL ADHESION MOLECULE (VCAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    84.
    发明申请
    RNA INTERFERENCE MEDIATED INHIBITION OF VASCULAR CELL ADHESION MOLECULE (VCAM) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 有权
    RNA干扰介导的血管细胞粘附分子（VCAM）基因表达的抑制使用短暂的核酸（siNA）

    公开(公告)号：US20090093436A1

    公开(公告)日：2009-04-09

    申请号：US12203055

    申请日：2008-09-02

    申请人： James McSwiggen ,  Leonid Beigelman

    发明人： James McSwiggen ,  Leonid Beigelman

    IPC分类号： A61K31/7088 ,  C07H21/00

    CPC分类号： C12N15/113 ,  C07H21/00 ,  C07H21/04 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3521

    摘要： This invention relates to compounds, compositions, and methods useful for modulating vascular cell adhesion molecule 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 vascular cell adhesion molecule 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 vascular cell adhesion molecule genes, such as vascular cell adhesion molecule-1 (VCAM-1).

    摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节血管细胞粘附分子基因表达的化合物，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通过RNA干扰（RNAi）的血管细胞粘附分子基因表达和/或活性的途径的其它基因的表达和活性的方法。 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） 用于调节血管细胞粘附分子基因表达的分子和方法，如血管细胞粘附分子-1（VCAM-1）。

    NUCLEIC ACID COMPOUNDS FOR INHIBITING APOB GENE EXPRESSION AND USES THEREOF
    85.
    发明申请
    NUCLEIC ACID COMPOUNDS FOR INHIBITING APOB GENE EXPRESSION AND USES THEREOF 审中-公开
    用于抑制APOB基因表达的核酸化合物及其用途

    公开(公告)号：US20080299659A1

    公开(公告)日：2008-12-04

    申请号：US12039668

    申请日：2008-02-28

    申请人： Steven C. Quay ,  James McSwiggen ,  Narendra K. Vaish ,  Mohammad Ahmadian

    发明人： Steven C. Quay ,  James McSwiggen ,  Narendra K. Vaish ,  Mohammad Ahmadian

    IPC分类号： C12N15/00 ,  C07H21/02

    CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/3231

    摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing ApoB gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to an ApoB mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an ApoB gene in a cell or in a subject to treat an ApoB-related disease.

    摘要翻译： 本公开提供能够降低或沉默ApoB基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其组合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与ApoB mRNA互补。 此外，meroduplex可以具有至少一个用5-甲基尿苷取代的尿苷，被锁定的核酸替代的核苷，或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中ApoB基因表达以治疗ApoB相关疾病的方法。

    NUCLEIC ACID COMPOUNDS FOR INHIBITING AKT GENE EXPRESSION AND USES THEREOF
    86.
    发明申请
    NUCLEIC ACID COMPOUNDS FOR INHIBITING AKT GENE EXPRESSION AND USES THEREOF 审中-公开
    用于抑制AKT基因表达的核酸化合物及其用途

    公开(公告)号：US20080293136A1

    公开(公告)日：2008-11-27

    申请号：US12039662

    申请日：2008-02-28

    申请人： Steven C. Quay ,  James McSwiggen ,  Narendra K. Vaish ,  Mohammad Ahmadian

    发明人： Steven C. Quay ,  James McSwiggen ,  Narendra K. Vaish ,  Mohammad Ahmadian

    IPC分类号： C12N5/06 ,  C07H21/04

    CPC分类号： C12N15/1137 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/53 ,  C12N2310/3521

    摘要： The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing AKT gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to an AKT mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an AKT gene in a cell or in a subject to treat an AKT-related disease.

    摘要翻译： 本公开提供能够降低或沉默AKT基因表达的meroduplex核糖核酸分子（mdRNA）。 本公开的mdRNA包含至少三条链，其结合形成由切口或间隙分开的至少两个不重叠的双链区域，其中一条链与AKT mRNA互补。 此外，meroduplex可以具有至少一个用5-甲基尿苷取代的尿苷，被锁定的核酸替代的核苷，或任选的其它修饰，以及它们的任何组合。 还提供减少细胞或受试者中AKT基因表达以治疗AKT相关疾病的方法。

    Rna interference mediated inhibition of nogo and nogo receptor gene expression using short interfering nucleic acid (sina)
    87.
    发明申请
    Rna interference mediated inhibition of nogo and nogo receptor gene expression using short interfering nucleic acid (sina) 审中-公开
    使用短干扰核酸（新生），Rna干扰介导的nogo和nogo受体基因表达的抑制

    公开(公告)号：US20070185043A1

    公开(公告)日：2007-08-09

    申请号：US10576690

    申请日：2004-08-20

    申请人： James McSwiggen ,  Bharat Chowrira ,  Peter Haeberli

    发明人： James McSwiggen ,  Bharat Chowrira ,  Peter Haeberli

    IPC分类号： A61K48/00 ,  C07H21/02

    CPC分类号： A61K49/0008 ,  C12N15/111 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/87 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/346 ,  C12N2310/53 ,  C12N2320/32 ,  C12N2330/30 ,  C12N2310/3521

    摘要： This invention relates to compounds, compositions, and methods useful for modulating NOGO and/or NOGO receptor 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 NOGO and/or NOGO receptor 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 NOGO and/or NOGO receptor genes, such as NOGO-A, NOGO-B, NOGO-C, NOGO-66 receptor, NI-35, NI-220, NI-250, myelin-associated glycoprotein, tenascin-R, and NG-2

    摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节NOGO和/或NOGO受体基因表达的化合物，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NOGO和/或NOGO受体基因表达和/或通过RNA干扰（RNAi）的活性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NOGO和/或NOGO受体基因如NOGO-A，NOGO-B，NOGO-C，NOGO-66受体，NI-35，NI-220，NI-250，髓磷脂 相关糖蛋白，腱生蛋白-R和NG-2

    RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
    88.
    发明申请
    RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA) 审中-公开

    公开(公告)号：US20070173473A1

    公开(公告)日：2007-07-26

    申请号：US11487788

    申请日：2006-07-17

    申请人： James McSwiggen ,  Vasant Jadhav ,  Narendra Vaish ,  Roberto Guerciolini ,  Chandra Vargeese

    发明人： James McSwiggen ,  Vasant Jadhav ,  Narendra Vaish ,  Roberto Guerciolini ,  Chandra Vargeese

    IPC分类号： A61K48/00 ,  C07H21/02

    CPC分类号： C12N15/1131 ,  C12N2310/14

    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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 Proprotein Convertase Subtilisin Kexin 9 (PCSK9) 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 PCSK9 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 hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits associated with PCSK9 gene expression or activity in a subject or organism.

    Rna interference mediated inhibition of cholesteryl ester transfer protein (cetp) gene expression using short interfering nucleic acid (sina)
    89.
    发明申请
    Rna interference mediated inhibition of cholesteryl ester transfer protein (cetp) gene expression using short interfering nucleic acid (sina) 审中-公开
    使用短干扰核酸（新生）的Rna干扰介导的胆固醇酯转移蛋白（cetp）基因表达的抑制

    公开(公告)号：US20070173467A1

    公开(公告)日：2007-07-26

    申请号：US10576751

    申请日：2004-08-19

    申请人： James McSwiggen ,  Barry Polisky

    发明人： James McSwiggen ,  Barry Polisky

    IPC分类号： A61K48/00 ,  C07H21/02

    CPC分类号： C12N15/113 ,  C12N2310/14

    摘要： This invention relates to compounds, compositions, and methods useful for modulating cholesteryl ester transfer protein (CETP) 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 CETP 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 CETP genes.

    摘要翻译： 本发明涉及使用短干扰核酸（siNA）分子调节胆固醇酯转移蛋白（CETP）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CETP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CETP基因表达的方法。