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    COMPETITIVE MODULATION OF MICRORNAS
    2.
    发明申请
    COMPETITIVE MODULATION OF MICRORNAS 有权
    MICRORNAS竞争性调制

    公开(公告)号：US20150299789A1

    公开(公告)日：2015-10-22

    申请号：US14651344

    申请日：2013-12-11

    申请人： ISIS PHARMACEUTICALS, INC.

    发明人： Xue-hai Liang ,  Stanley T. Crooke

    IPC分类号： C12Q1/68 ,  C12N15/113

    CPC分类号： C12Q1/6876 ,  A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2320/11 ,  C12Q2600/136 ,  C12Q2600/178

    摘要： The present invention provides compounds and methods for competitive modulation of microRNAs. Such compounds and methods have profound effects on cells. MicroRNAs (microRNAs), are small (approximately 18-24 nucleotides in length), non-coding RNA molecules encoded in the genomes of plants and animals. In certain instances, microRNAs regulate the expression of genes by binding to the 3′-untranslated regions (3′-UTR) of specific mRNAs. More than 1000 different microRNAs have been identified in plants and animals.

    摘要翻译： 本发明提供了用于微小RNA竞争调制的化合物和方法。 这些化合物和方法对细胞具有深远的影响。 微小RNA（microRNA）（小长度约为18-24个核苷酸），编码在植物和动物基因组中的非编码RNA分子。 在某些情况下，微小RNA通过结合特异性mRNA的3'非翻译区（3'-UTR）来调节基因的表达。 已经在植物和动物中鉴定了超过1000种不同的微小RNA。

    METHODS OF DETERMINING POTENCY OF CHEMICALLY-SYNTHESIZED OLIGONUCLEOTIDES
    3.
    发明申请
    METHODS OF DETERMINING POTENCY OF CHEMICALLY-SYNTHESIZED OLIGONUCLEOTIDES 审中-公开
    确定化学合成寡核苷酸的方法

    公开(公告)号：US20130252243A1

    公开(公告)日：2013-09-26

    申请号：US13724825

    申请日：2012-12-21

    申请人： ISIS PHARMACEUTICALS, INC.

    发明人： Walter F. Lima ,  Timothy Vickers ,  Stanley T. Crooke

    IPC分类号： C12Q1/68

    CPC分类号： C12Q1/6876 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2320/10 ,  Y10T436/143333

    摘要： Provided herein are methods for determining potency of RNAi agents. Such methods include, but are not limited to, cell-based and cell-free assays that measure binding of an RNAi agent with Ago2 or that measure Ago2 activity in the presence of such RNAi agents. Also provided are assays that determine potency of RNAi agents by assessing their ability to compete with other RNAi agents, including control RNAi agents, for binding and/or activation of Ago2.

    摘要翻译： 本文提供了确定RNAi剂效力的方法。 这样的方法包括但不限于测量RNAi剂与Ago2的结合或在这种RNAi剂存在下测量Ago2活性的基于细胞和无细胞的测定。 还提供了通过评估其与其他RNAi试剂（包括对照RNAi试剂）竞争与Ago2结合和/或激活的能力来测定RNAi试剂的效力的测定法。

    2'-METHOXY SUBSTITUTED OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN GENE MODULATIONS
    5.
    发明申请
    2'-METHOXY SUBSTITUTED OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN GENE MODULATIONS 有权
    用于基因调控的2'-甲氧基取代的低聚物和组合物

    公开(公告)号：US20150366894A1

    公开(公告)日：2015-12-24

    申请号：US14690120

    申请日：2015-04-17

    申请人： Isis Pharmaceuticals, Inc.

    发明人： Stanley T. Crooke

    IPC分类号： A61K31/713

    CPC分类号： A61K31/713 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/342 ,  C12N2310/344 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533

    摘要： Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2′-OCH3 substituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligomer has a modified sugar and/or backbone modification.

    摘要翻译： 提供包含第一和第二寡聚体的组合物，其中至少一部分第一寡聚体能够与至少一部分第二寡聚物杂交，至少一部分第一寡聚体与选定的靶互补并能够与选定的靶 核酸，并且第一或第二寡聚体中的至少一种包括修饰的糖和/或骨架修饰。 在一些实施方案中，修饰是糖部分上的2'-OCH 3取代基。 还提供低聚物/蛋白质组合物，其包含与选定的靶核酸互补并且能够与选定的靶核酸杂交的寡聚体和至少一种包含至少一部分RNA诱导的沉默复合物（RISC）的蛋白质，其中至少一个核苷酸 低聚物具有修饰的糖和/或骨架修饰。

    COMPOSITIONS AND METHODS FOR MODULATION OF LMNA EXPRESSION
    7.
    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

    申请人： Isis Pharmaceuticals, Inc.

    发明人： C. Frank Bennett ,  Kenneth W. Dobie ,  Susan M. Freier ,  Stanley T. Crooke

    IPC分类号： C12N15/113

    CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2320/33

    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。