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    RNAI CONSTRUCTS AND USES THEROF
    4.
    发明申请
    RNAI CONSTRUCTS AND USES THEROF 审中-公开
    RNAI构建和使用

    公开(公告)号：WO2010011346A1

    公开(公告)日：2010-01-28

    申请号：PCT/US2009004326

    申请日：2009-07-23

    申请人： RXI PHARMACEUTICALS CORP ,  SAMARSKY DMITRY ,  WOOLF TOD ,  SALOMON WILLIAM ,  KAMENS JOANNE ,  KHVOROVA ANASTASIA ,  PAVCO PAMELA A

    发明人： SAMARSKY DMITRY ,  WOOLF TOD ,  SALOMON WILLIAM ,  KAMENS JOANNE ,  KHVOROVA ANASTASIA ,  PAVCO PAMELA A

    IPC分类号： A61K31/712 ,  A61K31/713 ,  C12N15/11 ,  C12N15/113

    CPC分类号： C12N15/1137 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/531 ,  C12N2310/533 ,  C12N2320/51

    摘要： The invention relates to improved double-stranded RNAi constructs (sometimes referred to as "solo-rxRNA") and uses thereof. The construct comprises a structure formed in some aspects of the invention by two identical single-stranded polynucleotides, with the structure having two double-stranded stem regions (each having less than 21 base pairs) and a loop or bulge having about 4 to 11 nucleotides on each strand. The construct is resistant to cleavage by Dicer or other Dicer-like RNase III enzymes and is capable of being loaded into a RISC complex to effect RNA interference. In addition, the nucleotides of the present hairpin constructs may be modified to greatly enhance functionality, such as stability and specificity.

    摘要翻译： 本发明涉及改进的双链RNAi构建体（有时称为“solo-rxRNA”）及其用途。 该构建体包含在本发明的一些方面中由两个相同的单链多核苷酸形成的结构，该结构具有两个双链干区（每个具有少于21个碱基对）和具有约4至11个核苷酸的环或凸起 在每条链上。 该构建体对Dicer或其他类似切酶的RNA酶III酶的切割具有抗性，并且能够加载到RISC复合物中以实现RNA干扰。 另外，可以修饰本发明构建体的核苷酸以大大增强功能性，例如稳定性和特异性。

    APPARATUS AND SYSTEM HAVING DRY GENE SILENCING POOLS
    5.
    发明申请
    APPARATUS AND SYSTEM HAVING DRY GENE SILENCING POOLS 审中-公开
    具有干燥基因沉默池的装置和系统

    公开(公告)号：WO2006058046A3

    公开(公告)日：2006-09-28

    申请号：PCT/US2005042403

    申请日：2005-11-21

    申请人： DHARMACON INC ,  ROBERTSON BARBARA ,  LEAKE DEVIN ,  ROBINSON KATHRYN ,  MARSHALL WILLIAM S ,  KHVOROVA ANASTASIA

    发明人： ROBERTSON BARBARA ,  LEAKE DEVIN ,  ROBINSON KATHRYN ,  MARSHALL WILLIAM S ,  KHVOROVA ANASTASIA

    IPC分类号： C07H21/04 ,  C12N15/87

    CPC分类号： C12N15/87 ,  C12N15/111 ,  C12N2310/14 ,  C12N2320/11 ,  C12N2330/30

    摘要： A reverse transfection apparatus can be used for introducing siRNA into a cell to effect gene silencing. Such an apparatus can include a well plate having a well configured for transfecting cells. The well can include a substantially dry gene silencing composition that has at least two siRNAs which silences at least a first target gene. The gene silencing composition can be configured such that the at least two siRNAs are each capable of being solubilized or suspended in an aqueous medium in an amount sufficient for transfecting cells in the well. Additionally, the siRNAs can include a hairpin structure, modification, or a conjugate. Also, the at least siRNAs can be rationally designed. The reverse transfection apparatus can be provided as a kit or system that additionally includes cells, polynucleotide carriers, reverse transfection reagents, and the like.

    摘要翻译： 可以使用逆转染装置将siRNA导入细胞以实现基因沉默。 这种装置可以包括具有配置用于转染细胞的孔的孔板。 该孔可以包括基本干燥的基因沉默组合物，其具有沉默至少第一靶基因的至少两种siRNA。 可以配置基因沉默组合物，使得至少两种siRNA各自能够以足以转染孔中细胞的量溶解或悬浮于水性介质中。 另外，siRNA可以包含发夹结构，修饰或缀合物。 此外，至少可以合理设计siRNA。 逆转染设备可以作为另外包括细胞，多核苷酸载体，逆转染试剂等的试剂盒或系统提供。