DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES
    10.
    发明申请
    DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES 审中-公开
    用于有效和特异性靶向包含高度重复活性的DNA序列的切割内切酶的设计

    公开(公告)号:US20160273002A1

    公开(公告)日:2016-09-22

    申请号:US15031996

    申请日:2014-10-24

    申请人: CELLECTIS

    IPC分类号: C12N15/90 C07K14/195 C12N9/22

    摘要: The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.

    摘要翻译: 本发明在遗传编辑工具和基因工程方法领域。 它涉及设计用于在染色体中收缩高度重复动机的稀有内切核酸酶的工程,这些动机是某些遗传疾病的起源,特别是所谓的“三重重复疾病”,如亨廷顿疾病。 本发明包括用于收缩重复动机的方法,用于在重复病症的基因中重复动作的稀有内切核酸酶,编码它们的多核苷酸和载体以及所得药物组合物。