Compositions and methods for modulation of LMNA expression
    9.
    发明授权
    Compositions and methods for modulation of LMNA expression 有权
    LMNA表达调控的组成和方法

    公开(公告)号:US08791088B2

    公开(公告)日:2014-07-29

    申请号:US13566922

    申请日:2012-08-03

    IPC分类号: C12N15/11

    摘要: Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.

    摘要翻译: 本文公开了用于调节细胞,组织或动物中LMNA表达的化合物,组合物和方法。 还提供了目标验证的方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症的药物中的用途。 还提供了使用所公开的化合物鉴定所选mRNA的顺式剪接调节元件的方法。

    Modulation of prion expression
    10.
    发明授权
    Modulation of prion expression 有权
    调节朊病毒表达

    公开(公告)号:US08669102B2

    公开(公告)日:2014-03-11

    申请号:US13059058

    申请日:2009-08-14

    IPC分类号: C12N15/85 C07H21/04 C07H21/02

    CPC分类号: A61K31/7125

    摘要: Disclosed herein are compounds and methods for decreasing PrP and preventing, ameliorating, or treating a prion disease or conformational neurodegenerative disorder, in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to PrP include Creutzfeldt-Jakob disease (CJD); variant Creutzfeldt-Jakob Disease (vCJD); Gerstmann-Straussler-Scheinker syndrome; fatal familial insomnia; kuru; Bovine Spongiform Encephalopathy (BSE), e.g. “mad cow disease”; Chronic Wasting Disease (CWD); scrapie; transmissible mink encephalopathy; feline spongiform encephalopathy; ungulate spongiform encephalopathy; Alzheimer's disease; Parkinson's disease; Huntington's disease; and Amyotrophic Lateral Sclerosis (ALS).

    摘要翻译: 本文公开了在有需要的个体中减少PrP并预防,改善或治疗朊病毒疾病或构象神经变性疾病的化合物和方法。 通过施用针对PrP的反义化合物可以改善的疾病状况的实例包括克雅氏病(CJD); 变异克雅氏病(vCJD); Gerstmann-Straussler-Scheinker综合征; 致命性家族性失眠; 库鲁 牛海绵状脑病(BSE),例如。 “疯牛病”; 慢性疾病(CWD); scrap子 传染性貂脑病 猫海绵状脑病; 有蹄型海绵状脑病 阿尔茨海默氏病; 帕金森病 亨廷顿氏病 和肌萎缩性侧索硬化(ALS)。