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    GENE EDITING OF ANTICOAGULANTS
    1.
    发明申请
    GENE EDITING OF ANTICOAGULANTS 有权

    公开(公告)号：US20210317448A1

    公开(公告)日：2021-10-14

    申请号：US17262342

    申请日：2019-07-25

    Applicant: TOOLGEN INCORPORATED

    Inventor： Dong Woo SONG ,  Jung Min LEE ,  Kyu Jun LEE ,  Un Gi KIM

    IPC: C12N15/11 ,  A61P7/04 ,  A61K38/46 ,  A61K31/7088 ,  C12N9/22 ,  A61K9/00 ,  C12N15/86

    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.

    PLATFORM FOR EXPRESSING PROTEIN OF INTEREST IN LIVER
    5.
    发明申请
    PLATFORM FOR EXPRESSING PROTEIN OF INTEREST IN LIVER 有权

    公开(公告)号：US20210095316A1

    公开(公告)日：2021-04-01

    申请号：US16623017

    申请日：2018-06-15

    Applicant: TOOLGEN INCORPORATED

    Inventor： Seokjoong KIM ,  Dong Woo SONG ,  Kyu Jun LEE ,  Jung-Min LEE ,  Un-Gi KIM

    IPC: C12N15/90 ,  C12N9/64 ,  C12N15/11 ,  C12N9/22 ,  C12N15/86

    Abstract: Provided is a platform for expressing a protein of interest by artificially manipulating the liver, and more particularly, to a platform for alleviating or treating a genetic disorder or improving a body function by inducing expression by inserting a transgene (e.g., a therapeutic gene) which can function or be expressed normally, into a high-expression secretory gene, instead of a disease gene which functions or is expressed abnormally. The high-expression secretory gene includes the HP or APOC3 gene. The transgene includes one that is highly expressed using a promoter in a hepatocyte genome and is secretory out of the cell.

    COMPOSITION FOR TREATING HEMOPHILIA A BY CRISPR/CAS SYSTEM OF REVERTING FVIII GENE INVERSION
    7.
    发明申请
    COMPOSITION FOR TREATING HEMOPHILIA A BY CRISPR/CAS SYSTEM OF REVERTING FVIII GENE INVERSION 审中-公开

    公开(公告)号：US20200263204A1

    公开(公告)日：2020-08-20

    申请号：US16642348

    申请日：2018-09-28

    Applicant: TOOLGEN INCORPORATED

    Inventor： Seokjoong KIM ,  Dong Woo SONG ,  Kyu Jun LEE ,  Jung-Min LEE

    IPC: C12N15/90 ,  C12N9/22 ,  C07K14/755 ,  C12N15/86 ,  A61P7/04

    Abstract: The present invention relates to a CRISPR/Cas system having an inversion correction potential, which uses at least one guide RNA targeting a sequence region where two different homologs present on genomic introns are conjugated to each other in an inversion manner, and a Cas protein, and a CRISPR/Cas system of FVIII gene inversion correction potential that uses at least one guide RNA targeting an int22-1/3 homolog or int22-1/2 homolog sequence region present on intron 22 of coagulation factor VIII (F8) gene and a Cas protein. A CRISPR/Cas system according to the present invention comprises a system which employs a small-size Cas9 and a guide RNA fitted thereto, thereby enabling all CRISPR/Cas instruments to be easily packaged in one AAV, which is impossible in conventional large-size Cas9. In addition, the CRISPR/Cas system can induce normal gene expression thanks to the inversion gene correction potential thereof and is excellent as a technology capable of effectively overcoming the difficult intracellular delivery of large-size gene mutation through gene editing. Particularly, the system can induce normal FVIII expression by restoring the inversion of FVIII gene and thus is useful for the treatment of hemophilia A.

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