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40 条记录 (耗时 0.067 秒)

    RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
    4.
    发明公开
    RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
    通过RNA干扰介导的抑制前蛋白转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达的使用短干扰核酸（siNA）

    公开(公告)号：EP2052079A2

    公开(公告)日：2009-04-29

    申请号：EP07813032.5

    申请日：2007-07-17

    申请人： Sirna Therapeutics Inc.

    发明人： MCSWIGGEN, James ,  JADHAV, Vasant ,  VAISH, Narendra K. ,  GUERCIOLINI, Roberto ,  VARGEESE, Chandra

    IPC分类号： C12N15/11

    CPC分类号： C12N15/1137 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3521

    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 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 that are associated with gene expression or activity in a subject or organism.

    RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
    5.
    发明公开
    RNA INTERFERENCE MEDIATED INHIBITION OF STROMAL CELL-DERIVED FACTOR-1 (SDF-1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
    通过RNA干扰介导的来源的间质的基因的表达抑制因子1使用短干扰核酸（SDF-1）（新浪网）

    公开(公告)号：EP1891217A2

    公开(公告)日：2008-02-27

    申请号：EP06784507.3

    申请日：2006-05-26

    申请人： Sirna Therapeutics, Inc.

    发明人： GUERCIOLINI, Roberto ,  MCSWIGGEN, James ,  JADHAV, Vasant ,  VARGEESE, Chandra

    IPC分类号： C12N15/11 ,  C07K14/52

    CPC分类号： C12N15/1136 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3521

    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 stromal cell-derived factor- 1 (SDF-I) 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 stromal cell-derived factor- 1 (SDF-I) 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 stromal cell- derived factor- 1 (SDF-I) 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 SDF-I 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 angiogenesis related diseases traits and conditions, including but not limited to ocular disease, cancer and proliferative diseases, traits, and conditions, and/or other disease states, conditions, or traits associated with SDF-I gene expression or activity in a subject or organism.

    RNA INTERFERENCE MEDIATED INHIBITION OF MYC AND MYB GENES OR GENES OF THEIR RESPECTIVE PATHWAYS
    10.
    发明公开
    RNA INTERFERENCE MEDIATED INHIBITION OF MYC AND MYB GENES OR GENES OF THEIR RESPECTIVE PATHWAYS 审中-公开
    RNA干扰介导MYC和MYB基因或其相关途径基因的抑制作用

    公开(公告)号：EP1448590A2

    公开(公告)日：2004-08-25

    申请号：EP03709249.1

    申请日：2003-02-20

    申请人： Sirna Therapeutics, Inc.

    发明人： MCSWIGGEN, James ,  BEIGELMAN, Leonid

    IPC分类号： C07H21/04 ,  A61K48/00 ,  C12N15/00

    CPC分类号： C12N15/1135 ,  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 methods and reagents useful in modulating Myc and/or Myb gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to to 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 c-Myc, N-Myc, L-Myc, c-Myb, b-Myb, and v-Myb genes. The small nucleic acid molecules are useful in the treatment of cancer and other conditions, diseases and disorders.

    摘要翻译： 本发明涉及在各种应用中用于调节Myc和/或Myb基因表达的方法和试剂，包括用于治疗，诊断，靶标验证和基因组发现应用。 具体而言，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（miRNA）和短发夹RNA（shRNA） ）能够介导针对c-Myc，N-Myc，L-Myc，c-Myb，b-Myb和v-Myb基因的RNA干扰（RNAi）的分子。 小核酸分子可用于治疗癌症和其他病症，疾病和病症。