COMPOSITIONS AND METHODS FOR SILENCING EBOLA VIRUS GENE EXPRESSION
    1.
    发明申请
    COMPOSITIONS AND METHODS FOR SILENCING EBOLA VIRUS GENE EXPRESSION 审中-公开
    用于沉默EBOLA病毒基因表达的组合物和方法

    公开(公告)号:US20150111945A1

    公开(公告)日:2015-04-23

    申请号:US14229675

    申请日:2014-03-28

    摘要: The present invention provides compositions comprising therapeutic nucleic acids (e.g., interfering RNA such as siRNA) that target Ebola virus (EBOV) gene expression and methods of using such compositions to silence EBOV gene expression. More particularly, the invention provides unmodified and chemically modified interfering RNA which silence EBOV gene expression and methods of use thereof, e.g., for preventing or treating EBOV infections caused by one or more EBOV species such as Zaire EBOV. The invention also provides serum-stable nucleic acid-lipid particles comprising one or more interfering RNA molecules, a cationic lipid, and a non-cationic lipid, which can further comprise a conjugated lipid that inhibits aggregation of particles. Methods of silencing EBOV gene expression by administering one or more interfering RNA molecules to a mammalian subject are also provided.

    摘要翻译: 本发明提供包含针对埃博拉病毒(EBOV)基因表达的治疗性核酸(例如干扰RNA(例如siRNA))和使用这种组合物沉默EBOV基因表达的方法的组合物。 更具体地说,本发明提供了沉默EBOV基因表达的未修饰和化学修饰的干扰RNA及其使用方法,例如用于预防或治疗由一种或多种EBOV物种如扎伊尔EBOV引起的EBOV感染。 本发明还提供包含一种或多种干扰RNA分子,阳离子脂质和非阳离子脂质的血清稳定核酸 - 脂质颗粒,其可以进一步包含抑制颗粒聚集的共轭脂质。 还提供了通过向哺乳动物受试者施用一种或多种干扰RNA分子使EBOV基因表达沉默的方法。

    MODIFIED SIRNA MOLECULES AND USES THEREOF
    2.
    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:US20140179756A1

    公开(公告)日:2014-06-26

    申请号:US13952529

    申请日:2013-07-26

    IPC分类号: C12N15/113

    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。

    Modified siRNA molecules and uses thereof
    4.
    发明授权
    Modified siRNA molecules and uses thereof 有权
    修饰的siRNA分子及其用途

    公开(公告)号:US09074208B2

    公开(公告)日:2015-07-07

    申请号:US13952529

    申请日:2013-07-26

    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。

    NOVEL LIPID FORMULATIONS FOR DELIVERY OF THERAPEUTIC AGENTS TO SOLID TUMORS
    8.
    发明申请
    NOVEL LIPID FORMULATIONS FOR DELIVERY OF THERAPEUTIC AGENTS TO SOLID TUMORS 有权
    用于将治疗药物递送至固体肿瘤的新型脂质制剂

    公开(公告)号:US20150374834A1

    公开(公告)日:2015-12-31

    申请号:US14606905

    申请日:2015-01-27

    IPC分类号: A61K47/44 C12N15/113

    摘要: The present invention provides novel, serum-stable lipid particles comprising one or more active agents or therapeutic agents, methods of making the lipid particles, and methods of delivering and/or administering the lipid particles. More particularly, the present invention provides serum-stable nucleic acid-lipid particles (SNALP) comprising a nucleic acid (e.g., one or more interfering RNA molecules), methods of making the SNALP, and methods of delivering and/or administering the SNALP (e.g., for the treatment of cancer). In particular embodiments, the present invention provides tumor-directed lipid particles that preferentially target solid tumors. The tumor-directed formulations of the present invention are capable of preferentially delivering a payload such as a nucleic acid to cells of solid tumors compared to non-cancerous cells.

    摘要翻译: 本发明提供包含一种或多种活性剂或治疗剂的新型血清稳定脂质颗粒,制备脂质颗粒的方法,以及递送和/或施用脂质颗粒的方法。 更具体地说,本发明提供了包含核酸(例如一个或多个干扰RNA分子)的血清稳定的核酸 - 脂质颗粒(SNALP),制备SNALP的方法,以及递送和/或施用SNALP的方法 例如,用于治疗癌症)。 在具体实施方案中,本发明提供优先靶向实体瘤的肿瘤指导的脂质颗粒。 与非癌细胞相比,本发明的肿瘤引导制剂能够优先将有效载荷例如核酸递送至实体瘤细胞。