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2. 发明申请

WO2009082488A2 METHODS FOR CREATING AND IDENTIFYING FUNCTIONAL RNA INTERFERENCE ELEMENTS 审中-公开
标题翻译：用于创建和识别功能性RNA干扰元件的方法
公开(公告)号：WO2009082488A2
公开(公告)日：2009-07-02
申请号：PCT/US2008/014037
申请日：2008-12-23
申请人： BERGENBIO AS , MICKLEM, David , LORENS, James
发明人： MICKLEM, David , LORENS, James
IPC分类号： C12N15/11 , C12N15/10 , C12Q1/68
CPC分类号： C12N15/1086 , C12N15/10 , C12N15/111 , C12N15/64 , C12N15/66 , C12N2310/14 , C12N2320/10 , C12N2330/30 , C12Q1/6897
摘要： The invention relates to the control of gene expression. Specifically, the invention provides compositions and methods for the production and use of recombinant nucleic acid molecules that have the ability to specifically downregulate an expressed target gene in vivo . In some aspects, the invention provides methods for producing a hairpin DNA molecule where part of the molecule is derived from an mRNA that is a target for a small interfering RNA (siRNA) derived from the hairpin. In other aspects, the invention provides synthetic hairpin adapter oligonucleotides that are used in the construction of siRNA- producing cassettes. In other aspects, the invention provides methods for testing for the presence or absence of specific inhibitory activity of an RNAi trigger molecule, and in still other aspects, the invention provides methods for identifying an active RNAi trigger molecule from a library of RNAi trigger molecules. In still other aspects, the invention provides methods for identifying a polynucleotide from a plurality of candidate target polynucleotides that is specifically targeted by an RNAi trigger molecule. In other aspects, the invention provides epi-allelic series of hypomorphic RNAi trigger molecules specific for any gene of interest, where the series of RNAi trigger molecules have a variety of uses including analysis of gene function and drug target development.
摘要翻译：本发明涉及基因表达的控制。具体而言，本发明提供了生产和使用具有在体内特异性下调表达的靶基因的能力的重组核酸分子的组合物和方法。在一些方面，本发明提供了用于产生发夹DNA分子的方法，其中分子的一部分衍生自作为衍生自发夹的小干扰RNA（siRNA）的靶的mRNA。在其它方面，本发明提供用于构建产siRNA盒的合成发夹衔接寡核苷酸。在其他方面，本发明提供了用于测试RNAi触发分子的特异性抑制活性的存在或不存在的方法，并且在其它方面，本发明提供了从RNAi触发分子文库中鉴定活性RNAi触发分子的方法。在其它方面，本发明提供了从由RNAi触发分子专门靶向的多个候选靶多核苷酸中鉴定多核苷酸的方法。在其它方面，本发明提供了对于任何目的基因特异性的异型RNAi触发分子的等位基因系列，其中一系列RNAi触发分子具有各种用途，包括基因功能分析和药物靶标开发。

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