COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION
    9.
    发明申请
    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:US20140309282A1

    公开(公告)日:2014-10-16

    申请号:US14315186

    申请日:2014-06-25

    IPC分类号: C12N15/113

    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。