Transgenic reporter system that reveals expression profiles and regulation mechanisms of alternative splicing in mammalian organisms
    1.
    发明授权
    Transgenic reporter system that reveals expression profiles and regulation mechanisms of alternative splicing in mammalian organisms 有权
    转基因报告系统,揭示哺乳动物生物体内可变剪接的表达谱和调节机制

    公开(公告)号:US09273364B2

    公开(公告)日:2016-03-01

    申请号:US13700890

    申请日:2011-05-31

    CPC分类号: C12Q1/6897 C12N15/1086

    摘要: An object of the present invention is to develop a new alternative splicing reporter system and to provide a method for detecting alternative splicing patterns in a mammalian multicellular organism more precisely, a method for identifying efficiently substances and gene regions that affect alternative splicing in a mammalian multicellular organism, and the like by utilizing the alternative splicing reporter system. Specifically, the present invention relates to a method for detecting alternative splicing in a mammalian multicellular organism, and a method for identifying substances and gene regions that affect alternative splicing in a mammalian multicellular organism, which use a DNA construct in which at least two different reporter genes are inserted into a specific gene that undergoes alternative splicing, or a combination of DNA constructs (a combination of at least two different DNA constructs) in which DNA construct a reporter gene is inserted into a specific gene that undergoes alternative splicing.

    摘要翻译: 本发明的一个目的是开发一种新的选择性剪接报道系统,并提供一种用于更准确地检测哺乳动物多细胞生物体中的选择性剪接模式的方法,一种用于有效识别影响哺乳动物多细胞中的选择性剪接的物质和基因区域的方法 生物体等通过利用可变剪接报告系统。 具体地,本发明涉及用于检测哺乳动物多细胞生物体中的选择性剪接的方法,以及用于鉴定影响哺乳动物多细胞生物体中的选择性剪接的物质和基因区域的方法,其使用DNA构建体,其中至少两个不同的报道子 将基因插入到经历可选剪接的特定基因中,或将DNA构建体报告基因插入经历可选剪接的特定基因的DNA构建体(至少两种不同DNA构建体的组合)的组合中。

    Prophylactic or ameliorating agent for genetic diseases
    2.
    发明授权
    Prophylactic or ameliorating agent for genetic diseases 有权
    遗传疾病预防或改善剂

    公开(公告)号:US09241929B2

    公开(公告)日:2016-01-26

    申请号:US13805151

    申请日:2011-06-27

    摘要: An object of the present invention is to provide a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation. The prophylactic or ameliorating agent used in the present invention is a prophylactic or ameliorating agent for a genetic disease that is caused by a mutation in an exon of a gene and is capable of forming a functional truncated protein by skipping of the exon comprising the mutation, wherein the prophylactic or ameliorating agent contains a compound having a molecular weight of 1500 or lower.

    摘要翻译: 本发明的目的是提供由基因外显子突变引起的遗传疾病的预防或改善剂,并且能够通过跳过包含突变的外显子形成功能性截短的蛋白质。 用于本发明的预防或改善剂是由基因外显子突变引起的遗传疾病的预防或改善剂,能够通过跳过包含突变的外显子形成功能性截短蛋白, 其中所述预防或改善剂含有分子量为1500以下的化合物。