首页> 外国专利> iPS CELL GROUP HAVING DIFFERENT MITOCHONDRIAL DISEASE INDUCED VARIANT mtDNA RATIO

iPS CELL GROUP HAVING DIFFERENT MITOCHONDRIAL DISEASE INDUCED VARIANT mtDNA RATIO

机译:iPS细胞群具有不同的线粒体疾病诱发的变异mtDNA比率

摘要

PROBLEM TO BE SOLVED: To provide a tool that is useful for clarifying pathosis mechanism of a mitochondrial disease caused by abnormality of mitochondria DNA and developing a treatment technique thereof.SOLUTION: A kit contains two types or more of iPS cell populations in which ratio of variant mtDNA having mitochondrial disease induced variation is different, and which is derived from the same iPS cell clone. A kit contains at least one type selected from the group consisting of TAL effector nuclease in which RVD is HD-NG-NI-HD-NG-HD-HD-NI-NI-NI-NN-NI-HD-HD-NI-HD from an N terminal side, mRNA of the TAL effector nuclease, and an expression cassette of the TAL effector nuclease, and at least one type selected from the group consisting of and TAL effector nuclease having other RVD, mRNA of the TAL effector nuclease, and the expression cassette of the TAL effector nuclease.SELECTED DRAWING: None
机译:解决的问题:提供一种用于阐明由线粒体DNA异常引起的线粒体疾病的致病机理并开发其治疗技术的工具。解决方案:试剂盒包含两种或两种以上iPS细胞群,其中具有线粒体疾病诱导变异的mtDNA变异体是不同的,并且源自同一iPS细胞克隆。试剂盒包含至少一种选自TAL效应子核酸酶的药物,其中RVD为HD-NG-NI-HD-NG-HD-HD-HD-NI-NI-NI-NN-NI-HD-HD-NI-从N末端侧看的HD,TAL效应子核酸酶的mRNA和TAL效应子核酸酶的表达盒,以及选自具有其他RVD的TAL效应子核酸酶和TAL效应子核酸酶的mRNA中的至少一种,以及TAL效应子核酸酶的表达盒。

著录项

  • 公开/公告号JP2018139580A

    专利类型

  • 公开/公告日2018-09-13

    原文格式PDF

  • 申请/专利权人 FUJITA GAKUEN;

    申请/专利号JP20170199578

  • 发明设计人 HACHIMAN NAOKI;HATA RYUJI;OMI MINORU;

    申请日2017-10-13

  • 分类号C12N5/10;C12N9/16;C12N15/09;A61P21/00;A61P43/00;A61P9/00;A61P25/08;A61P27/02;A61P25/28;A61P27/16;A61P3/10;A61K35/545;

  • 国家 JP

  • 入库时间 2022-08-21 13:14:34

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