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    COMPOSITIONS AND METHODS FOR INHIBITING GENE SILENCING BY RNA INTERFERENCE
    4.
    发明申请
    COMPOSITIONS AND METHODS FOR INHIBITING GENE SILENCING BY RNA INTERFERENCE 审中-公开
    通过RNA干扰抑制基因沉默的组合物和方法

    公开(公告)号：WO2007095387A2

    公开(公告)日：2007-08-23

    申请号：PCT/US2007/004223

    申请日：2007-02-16

    申请人： DHARMACON, INC. ,  VERMEULEN, Annaleen ,  ROBERTSON, Barbara ,  BASKERVILLE, Scott ,  YAMADA, Christina ,  LEAKE, Devin ,  FEDOROV, Yuriy ,  KARPILOW, Jon ,  KHVOROVA, Anastasia

    发明人： VERMEULEN, Annaleen ,  ROBERTSON, Barbara ,  BASKERVILLE, Scott ,  YAMADA, Christina ,  LEAKE, Devin ,  FEDOROV, Yuriy ,  KARPILOW, Jon ,  KHVOROVA, Anastasia

    CPC分类号： C12N15/111 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2310/53 ,  C12N2320/50 ,  C12N2310/3521

    摘要： The present invention provides compositions and methods for inhibiting gene silencing by the RNAi pathway. The RNAi inhibitors of the invention have a reverse complement (RC) region to the target molecule of interest (e.g., miRNA) in association with at least one flanking region coupled to either at the 3' or 5' end of the RC region. The flanking regions can be single-stranded or can have one or more regions of double stranded nucleic acid with or without a hairpin loop. The RNAi inhibitors described herein can inhibit endogenous targets, including but not limited to microRNAs, or piRNAs, or can be used to inhibit the effects of exogenously introduced molecules, such as synthetic siRNAs, siRNAs expressed from vector constructs (e.g., viral expression systems), or siRNAs generated by enzymatic methods. Inhibition is specific, potent, prolonged, and can be performed on a single target or multiple targets simultaneously.

    摘要翻译： 本发明提供了通过RNAi途径抑制基因沉默的组合物和方法。 本发明的RNAi抑制剂与至少一个与RC区3'端或5'末端连接的侧翼区相关联，具有与目的靶分子（例如miRNA）的反向互补序列（RC）区。 侧翼区可以是单链的，或者可以具有一个或多个具有或不具有发夹环的双链核酸区域。 本文所述的RNAi抑制剂可抑制内源性靶标，包括但不限于微小RNA或piRNA，或可用于抑制外源引入分子（如合成siRNAs），由载体构建体表达的siRNA（例如病毒表达系统） ，或通过酶法产生的siRNA。 抑制是具体的，有效的，延长的，并且可以同时在单个目标或多个靶上进行。

    ENHANCED BIOTHERAPEUTIC PRODUCTION USING INHIBITORY RNA
    7.
    发明申请
    ENHANCED BIOTHERAPEUTIC PRODUCTION USING INHIBITORY RNA 审中-公开
    使用抑制性RNA增强生物制剂生产

    公开(公告)号：WO2009012173A2

    公开(公告)日：2009-01-22

    申请号：PCT/US2008069859

    申请日：2008-07-11

    申请人： DHARMACON INC ,  LADINE JAMES R ,  MARSHALL WILLIAM S ,  FEDOROV YURIY ,  YAMADA CHRISTINA

    发明人： LADINE JAMES R ,  MARSHALL WILLIAM S ,  FEDOROV YURIY ,  YAMADA CHRISTINA

    IPC分类号： C12P21/00 ,  A61K38/00 ,  C12N15/09

    CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3515 ,  C12N2310/3521

    摘要： Compositions, kits, systems, equipment, and protocols utilize synthetic siRNA having a delivery facilitating moiety in improved bioprocesses that enhance the production of biomaterials. The siRNA can target genes associated with the following: 1) deleterious vector derived genes; 2) genes that confer non-optimal growth or differentiation properties to the cells; 3) genes that can influence heterogeneity or post-translational modification pattern of the desirable gene product; 4) genes that highly express non-desired proteins; 5) genes that express proteins which interfere with purification of the desired protein; and 6) other genes that can interfere with the bioprocess.

    摘要翻译： 组合物，试剂盒，系统，设备和方案利用具有递送促进部分的合成siRNA改进生物过程以增强生物材料的生产。 siRNA可靶向与以下相关的基因：1）有害的载体衍生基因; 2）赋予细胞非最佳生长或分化特性的基因; 3）可影响期望基因产物的异质性或翻译后修饰模式的基因; 4）高度表达非期望蛋白质的基因; 5）表达干扰所需蛋白质纯化的蛋白质的基因; 和6）其他可能干扰生物过程的基因。