GENE EDITING OF ANTICOAGULANTS
    3.
    发明申请

    公开(公告)号:US20210317448A1

    公开(公告)日:2021-10-14

    申请号:US17262342

    申请日:2019-07-25

    Abstract: The present invention relates to a composition for gene manipulation for artificially manipulating a blood coagulation inhibitory gene present in the genome of a cell to regulate a blood coagulation system. More particularly, the present invention relates to a composition for gene manipulation, which includes a guide nucleic acid capable of targeting a blood coagulation inhibitory gene, and an editor protein. Also, the present invention relates to a method of treating or improving coagulopathy using the composition for gene manipulation for artificially manipulating a blood coagulation inhibitory gene.

    ARTIFICIALLY ENGINEERED SC FUNCTION CONTROL SYSTEM

    公开(公告)号:US20190276503A1

    公开(公告)日:2019-09-12

    申请号:US16349672

    申请日:2017-09-28

    Abstract: The present invention relates to an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder, and to a use thereof. More specifically, the present invention relates to a system capable of performing artificial SC function control and/or treating or alleviating a disease due to an SC function disorder, the system comprising: an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder; and/or a composition for treating or alleviating a disease due to an SC function disorder. In a specific aspect, the present invention relates to an SC function-controlling system by an SC function-controlling factor, such as artificially manipulated PMP22, and/or an expression product thereof.

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