COMPOSITIONS AND METHODS FOR SILENCING SMAD4
    7.
    发明申请
    COMPOSITIONS AND METHODS FOR SILENCING SMAD4 有权
    用于沉默SMAD4的组合物和方法

    公开(公告)号:US20130172405A1

    公开(公告)日:2013-07-04

    申请号:US13725796

    申请日:2012-12-21

    IPC分类号: C07H21/02 A61K48/00

    摘要: The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA (e.g., dsRNA such as siRNA) that target SMAD4 gene expression, lipid particles comprising one or more (e.g., a cocktail) of the therapeutic nucleic acids, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles (e.g., for treating anemia of inflammation in humans).

    摘要翻译: 本发明提供了包含靶向SMAD4基因表达的治疗性核酸例如干扰RNA(例如dsRNA例如siRNA)的组合物,包含治疗性核酸的一种或多种(例如混合物)的脂质颗粒,制备该脂质的方法 颗粒和递送和/或施用脂质颗粒的方法(例如,用于治疗人类的炎症性贫血)。

    MODIFIED SIRNA MOLECULES AND USES THEREOF
    9.
    发明申请
    MODIFIED SIRNA MOLECULES AND USES THEREOF 有权
    改性SIRNA分子及其用途

    公开(公告)号:US20120328668A1

    公开(公告)日:2012-12-27

    申请号:US13457394

    申请日:2012-04-26

    摘要: The present invention provides chemically modified siRNA molecules and methods of using such siRNA molecules to silence target gene expression. Advantageously, the modified siRNA of the present invention is less immunostimulatory than its corresponding unmodified siRNA sequence and retains RNAi activity against the target sequence. The present invention also provides nucleic acid-lipid particles comprising a modified siRNA, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. The present invention further provides methods of silencing gene expression by administering a modified siRNA to a mammalian subject. Methods for identifying and/or modifying an siRNA having immunostimulatory properties are also provided.

    摘要翻译: 本发明提供化学修饰的siRNA分子和使用这种siRNA分子沉默靶基因表达的方法。 有利地,本发明的修饰的siRNA比其相应的未修饰的siRNA序列具有更少的免疫刺激,并保留针对靶序列的RNAi活性。 本发明还提供了包含修饰的siRNA,阳离子脂质和非阳离子脂质的核酸 - 脂质颗粒,其可进一步包含抑制颗粒聚集的共轭脂质。 本发明还提供了通过向哺乳动物受试者施用修饰的siRNA来沉默基因表达的方法。 还提供了用于鉴定和/或修饰具有免疫刺激性质的siRNA的方法。