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    ENDONUCLEASE TARGETING BLOOD COAGULATION FACTOR VIII GENE AND COMPOSITION FOR TREATING HEMOPHILIA COMPRISING SAME
    4.
    发明公开
    ENDONUCLEASE TARGETING BLOOD COAGULATION FACTOR VIII GENE AND COMPOSITION FOR TREATING HEMOPHILIA COMPRISING SAME 审中-公开
    核酸内切酶凝血因子VIII基因和组合物，用于治疗包含相同血凝素的血友病

    公开(公告)号：EP3243529A2

    公开(公告)日：2017-11-15

    申请号：EP16735167.5

    申请日：2016-01-06

    Applicant: Industry-Academic Cooperation Foundation Yonsei University ,  Institute for Basic Science

    Inventor： KIM, Dong Wook ,  KIM, Jin Soo ,  PARK, Chul Yong ,  KIM, Duk Hyoung ,  KIM, Jung Eun ,  KWEON, Ji Yeon

    IPC: A61K48/00 ,  C12N15/113 ,  C07K14/755 ,  C12N5/074 ,  A61K35/12 ,  C12N9/16

    Abstract: The present invention provides a method for inducing an inversion of normal blood coagulation factor VIII (F8) gene, a method for correcting an inversion of blood coagulation factor VIII gene in which the inversion has occurred, and a Hemophilia A patient-derived induced pluripotent stem cell in which the inversion is corrected, constructed using the same. The method of the present invention effectively reproduces the inversion of intron 1 and intron 22 of the F8 gene, which is responsible for the majority of severe hemophilia A, and thereby may be effectively used for studying the development mechanism of hemophilia A and as a research tool for screening therapeutic agents. The inversion-corrected induced pluripotent stem cell constructed according the method of the present invention enables an efficient and fundamental treatment for hemophilia A by restoring a genotype in which mutation has occurred to a wild type-like state, without limitation via normal gene or protein delivery.

    Abstract translation: 本发明提供一种诱导正常凝血因子VIII（F8）基因反转的方法，校正发生了倒位的凝血因子VIII基因的倒位的方法和血友病A患者衍生的诱导多能干 反转被修正的单元，使用它来构造。 本发明的方法有效地再现了导致大多数严重血友病A的F8基因的内含子1和内含子22的倒位，并且因此可以有效地用于研究血友病A的发展机制并且作为研究 用于筛选治疗剂的工具。 根据本发明的方法构建的反转校正的诱导性多能干细胞通过将发生突变的基因型恢复至野生型样状态而无限制地通过正常的基因或蛋白质递送而实现对血友病A的有效且基本的治疗 。

    METHOD FOR REGULATING GENE EXPRESSION USING CAS9 PROTEIN EXPRESSED FROM TWO VECTORS
    6.
    发明公开
    METHOD FOR REGULATING GENE EXPRESSION USING CAS9 PROTEIN EXPRESSED FROM TWO VECTORS 审中-公开
    用两种载体表达的CAS9蛋白调节基因表达的方法

    公开(公告)号：EP3222728A1

    公开(公告)日：2017-09-27

    申请号：EP15861198.8

    申请日：2015-11-19

    Applicant: Institute for Basic Science

    Inventor： KIM, Jin Soo ,  KOO, Tae Young

    IPC: C12N15/63 ,  C12N9/16

    CPC classification number: C12N15/746 ,  C12N9/16 ,  C12N9/22 ,  C12N15/11 ,  C12N15/63 ,  C12N15/8645 ,  C12N15/867

    Abstract: The present invention relates to a method for regulating gene expression, comprising introducing into a cell each of a recombinant vector which expresses a first domain comprising N-terminus of a Cas9 protein, and a recombinant vector which expresses a second domain comprising C-terminus of a Cas9 protein, a composition comprising the recombinant vectors, a kit for regulating gene expression, and a method for intracellular production of Cas9 protein. Moreover, the present invention relates to a transformed cell introduced with a viral vector which packages the first domain, and a viral vector which packages the second domain, and to a composition comprising a virus produced therefrom.

    Abstract translation: 本发明涉及调节基因表达的方法，包括向细胞中导入表达包含Cas9蛋白的N端的第一结构域的重组载体和表达第二结构域的重组载体，所述第二结构域包含 Cas9蛋白质，包含该重组载体的组合物，用于调节基因表达的试剂盒以及用于细胞内产生Cas9蛋白质的方法。 此外，本发明涉及导入了包装第一结构域的病毒载体和包装第二结构域的病毒载体的转化细胞，以及包含由其产生的病毒的组合物。

    METHOD FOR DETECTING OFF-TARGET SITE OF GENETIC SCISSORS IN GENOME
    7.
    发明公开
    METHOD FOR DETECTING OFF-TARGET SITE OF GENETIC SCISSORS IN GENOME 审中-公开
    在基因组中检测遗传剪刀关闭目标点的方法

    公开(公告)号：EP3219810A1

    公开(公告)日：2017-09-20

    申请号：EP15858194.2

    申请日：2015-11-13

    Applicant: Institute for Basic Science

    Inventor： KIM, Jin Soo ,  KIM, Dae Sik ,  BAE, Sang Su

    IPC: C12Q1/68 ,  C12N9/22 ,  G06F19/22

    CPC classification number: C12N9/22 ,  C12Q1/68 ,  G06F19/22 ,  Y02A50/451

    Abstract: The present disclosure relates to a method for detecting off-target sites of a programmable nuclease in a genome, and specifically, to a method for detecting off-target sites through data analysis by subjecting the genome isolated in vitro to programmable nucleases to cleave the genome and then performing whole genome sequencing or deep sequencing, and to a method for selecting on-target sites of a programmable nuclease, which minimizes the off-target effect, using this method. The Digenome-seq of the present disclosure can detect the off-target sites of a programmable nuclease on the genomic scale at a high degree of reproducibility, and thus can be used in the manufacture of programmable nucleases having high target specificity and the study thereof.

    Abstract translation: 本发明涉及用于检测基因组中可编程核酸酶的脱靶位点的方法，具体涉及通过数据分析检测脱靶位点的方法，所述方法通过使体外分离的基因组经受可程序化的核酸酶以切割基因组 然后进行全基因组测序或深度测序，以及使用该方法选择使可脱靶效应最小化的可编程核酸酶的在靶位点的方法。 本公开的Digenome-seq可以高度重现性地在基因组规模上检测可编程核酸酶的脱靶位点，并因此可用于制备具有高靶特异性的可编程核酸酶及其研究。

    METHOD FOR SENSITIVE DETECTION OF TARGET DNA USING TARGET-SPECIFIC NUCLEASE
    9.
    发明公开
    METHOD FOR SENSITIVE DETECTION OF TARGET DNA USING TARGET-SPECIFIC NUCLEASE 审中-公开
    VERFAHREN ZUR EMPFINDLICHEN DETEKTION VON ZIEL-DNA MIT ZIELSPEZIFISCHER NUKLEASE

    公开(公告)号：EP3150718A1

    公开(公告)日：2017-04-05

    申请号：EP15800090.1

    申请日：2015-05-28

    Applicant: Toolgen Incorporated ,  Institute for Basic Science

    Inventor： KIM, Jin Soo ,  KIM, So Jung ,  LEE, Seung Hwan ,  KIM, Seok Joong

    IPC: C12Q1/68 ,  G01N33/574 ,  G01N33/53

    CPC classification number: C12Q1/6844 ,  C12Q1/68 ,  C12Q2521/301 ,  G01N33/53 ,  G01N33/574

    Abstract: The present invention relates to a method for analyzing a genotype using a target-specific nuclease and, specifically, to a method for diagnosing cancer or analyzing a genotype by removing wild type DNA or particular genotype DNA using a target-specific nuclease or a variant thereof to amplify or concentrate only a small amount of DNA which has a difference in variation, such as a mutation, or genotype, and to a method for separating target DNA sesusing a target-specific nuclease or a variant thereof. Such methods are novel paradigm methods contrary to existing simple target-specific nucleases for post-PCR recognition of normal genotype and carcinogenic genotype, and can be favorably used in the early diagnosis of cancer or analysis of similar genotypes.

    Abstract translation: 本发明涉及使用靶特异性核酸酶分析基因型的方法，具体地，涉及用于诊断癌症的方法或通过使用靶特异性核酸酶或其变体除去野生型DNA或特定基因型DNA来分析基因型的方法 扩增或浓缩仅具有变异差异（如突变或基因型）的少量DNA，以及分离靶标特异性核酸酶或其变体的靶DNA的分离方法。 这种方法是与现有的简单目标特异性核酸酶相反的新型范式方法，用于正常基因型和致癌基因型的PCR后识别，可以有利地用于癌症的早期诊断或类似基因型的分析。

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