RNA INTERFERENCE MEDIATED INHIBITION OF PROTEIN TYROSINE PHOSPHATASE-1B (PTP-1B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
    44.
    发明申请
    RNA INTERFERENCE MEDIATED INHIBITION OF PROTEIN TYROSINE PHOSPHATASE-1B (PTP-1B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) 审中-公开
    RNA干扰介导的抑制蛋白酪氨酸磷酸化酶-1B(PTP-1B)基因表达使用短暂的核酸(siNA)

    公开(公告)号:US20090239931A1

    公开(公告)日:2009-09-24

    申请号:US11748029

    申请日:2007-05-14

    IPC分类号: A61K31/7105

    摘要: This invention relates to compounds, compositions, and methods useful for modulating protein tyrosine phosphatase-1B (PTP-1B) 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 PTP-1B 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 PTP-1B genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing obesity, insulin resistance, diabetes (eg. type II and type I diabetes) in a subject or organism, and for any other disease, trait, or condition that is related to or will respond to the levels of PTP-1B 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)分子调节蛋白酪氨酸磷酸酶-1B(PTP-1B)基因表达的化合物,组合物和方法。 本发明还涉及使用小核酸分子调节涉及PTP-1B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PTP-1B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内的肥胖,胰岛素抵抗,糖尿病(例如II型和I型糖尿病)和任何其它疾病,性状, 或与细胞或组织中PTP-1B水平相关或将会对其单独或与其它治疗或疗法组合相关的病症。

    RNA interference mediated inhibition of sterol regulatory element binding protein 1 (SREBP1) gene expression using short interfering nucleic acid (siNA)

    公开(公告)号:US20080249040A1

    公开(公告)日:2008-10-09

    申请号:US11488374

    申请日:2006-07-18

    IPC分类号: A61K31/70 C07H21/00 A61P43/00

    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 Sterol Regulatory Element-Binding Protein 1 (SREBP1) 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 Sterol Regulatory Element-Binding Protein 1 (SREBP1) 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 Sterol Regulatory Element-Binding Protein 1 (SREBP1) 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 SREBP1 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 SREBP1 gene expression or activity in a subject or organism.

    RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
    49.
    发明申请
    RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor 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)抑制血管内皮生长因子和血管内皮生长因子受体基因表达

    公开(公告)号:US20050233998A1

    公开(公告)日:2005-10-20

    申请号:US10944611

    申请日:2004-09-16

    CPC分类号: C12N15/113 C12N2310/14

    摘要: This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR 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 VEGF and/or VEGFR 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 VEGF and/or VEGFR genes.

    摘要翻译: 本发明涉及使用短干扰核酸(siNA)分子调节VEGF和/或VEGFR基因表达的化合物,组合物和方法。 本发明还涉及使用小核酸分子调节参与VEGF和/或VEGFR基因表达和/或活性通过RNA干扰(RNAi)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VEGF和/或VEGFR基因表达的方法。

    Inhibition of gene expression using duplex forming oligonucleotides
    50.
    发明申请
    Inhibition of gene expression using duplex forming oligonucleotides 审中-公开
    使用双链体形成寡核苷酸抑制基因表达

    公开(公告)号:US20050233329A1

    公开(公告)日:2005-10-20

    申请号:US10727780

    申请日:2003-12-03

    IPC分类号: C07H21/04 C12Q1/68

    摘要: The present invention concerns methods and nucleic acid based reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, veterinary, agricultural, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to double strand forming oligonucleotides (DFO) that can self assemble to form double stranded oligonucleotides, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA) molecules, and modulate gene expression, for example by RNA interference (RNAi). The self complementary DFO nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

    摘要翻译: 本发明涉及可用于调节各种应用中的基因表达的方法和基于核酸的试剂,包括在治疗,兽医,农业,诊断,靶标验证和基因组发现应用中的用途。 具体地,本发明涉及可以自组装形成双链寡核苷酸的双链形成寡核苷酸(DFO),例如短干扰核酸(siNA),短干扰RNA(siRNA)分子和调节基因表达,例如通过RNA 干扰(RNAi)。 自身互补的DFO核酸分子可用于治疗对细胞,组织或生物中的基因表达或活性的调节作出反应的任何疾病或病症。