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.

公开(公告)号：US20200277630A1

公开(公告)日：2020-09-03

申请号：US16650993

申请日：2018-09-28

Applicant: TOOLGEN INCORPORATED ,  SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION ,  SEOUL NATIONAL UNIVERSITY HOSPITAL

Inventor： Dong Woo SONG ,  Jung Min LEE ,  Un Gi KIM ,  Jeong Hun KIM ,  Dong Hyun JO

IPC: C12N15/86 ,  C12N15/113 ,  C12N9/22 ,  A61P27/02

Abstract: The present invention relates to a composition for gene manipulation for treating or improving a retinal dysfunction disease or a method using the same. More particularly, the present invention relates to a composition for gene manipulation including a guide nucleic acid capable of targeting a retinal function-forming gene and a method of treating or improving a disease caused by retinal dysfunction by artificially manipulating and/or correcting a retinal function-forming gene using the same.

公开(公告)号：US20250115906A1

公开(公告)日：2025-04-10

申请号：US18727149

申请日：2023-01-12

Applicant: TOOLGEN INCORPORATED

Inventor： Dong Woo SONG ,  Hyerim LEE ,  Hye-Kyung OH ,  Beom Seok CHOI ,  Kyu Jun LEE ,  Nan Yeong GO ,  Jae Young LEE

IPC: C12N15/113 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86

Abstract: The present invention relates to a Schwann cell-specific promoter and use thereof. More particularly, the present invention relates to an artificially engineered minimal promoter that specifically operates in Schwann cells, wherein the artificially engineered minimal promoter is derived from an MPZ (Myelin Protein Zero or PO) promoter. In addition, the present invention relates to a vector using the Schwann cell-specific promoter and a method for treating Schwann cell-related diseases using same.

公开(公告)号：US20210254054A1

公开(公告)日：2021-08-19

申请号：US17236660

申请日：2021-04-21

Applicant: TOOLGEN INCORPORATED ,  SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION ,  SEOUL NATIONAL UNIVERSITY HOSPITAL

Inventor： Jeong Hun KIM ,  Sung Wook PARK ,  Seokjoong KIM ,  Dong Woo SONG

IPC: C12N15/11 ,  C12N9/22 ,  A61K48/00 ,  C12N15/113 ,  C07K14/47 ,  C07K14/52 ,  C07K14/515 ,  C12N15/86 ,  A61K9/00 ,  A61K38/46

Abstract: The present invention relates to an artificially manipulated neovascularization-associated factor for regulating neovascularization and a use thereof. More particularly, the present invention relates to a system for artificially regulating neovascularization, which includes an artificially manipulated neovascularization-associated factor for regulating neovascularization and/or a composition for artificially manipulating the neovascularization-associated factor. In a specific aspect, a neovascularization regulatory system including a neovascularization-associated factor such as artificially manipulated VEGFA, HIF1A, ANGPT2, EPAS1, or ANGPTL4 and/or an expression product thereof is provided.

公开(公告)号：US20180237771A1

公开(公告)日：2018-08-23

申请号：US15953141

申请日：2018-04-13

Applicant: TOOLGEN INCORPORATED ,  SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION ,  SEOUL NATIONAL UNIVERSITY HOSPITAL

Inventor： Jeong Hun KIM ,  Sung Wook PARK ,  Seokjoong KIM ,  Dong Woo SONG

IPC: C12N15/11 ,  C12N9/22 ,  C12N15/86 ,  A61K38/46 ,  A61K9/00

Abstract: The present invention relates to an artificially manipulated neovascularization-associated factor for regulating neovascularization and a use thereof. More particularly, the present invention relates to a system for artificially regulating neovascularization, which includes an artificially manipulated neovascularization-associated factor for regulating neovascularization and/or a composition for artificially manipulating the neovascularization-associated factor. In a specific aspect, a neovascularization regulatory system including a neovascularization-associated factor such as artificially manipulated VEGFA, HIF1A, ANGPT2, EPAS1, or ANGPTL4 and/or an expression product thereof is provided.

公开(公告)号：US20250051749A1

公开(公告)日：2025-02-13

申请号：US18723085

申请日：2022-12-23

Applicant: TOOLGEN INCORPORATED ,  SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION

Inventor： Su Cheong YEOM ,  Dong Woo SONG ,  Seokjoong KIM ,  Hye Kyung OH

IPC: C12N9/64 ,  C12N15/85

Abstract: The present application relates to a novel promoter and a use thereof. In one embodiment of the present application, provided is a nucleic acid construct comprising a novel promoter and a transgene of the present application. At this time, on the nucleic acid construct, the novel promoter of the present application can be operably linked to the transgene.

公开(公告)号：US20230287461A1

公开(公告)日：2023-09-14

申请号：US18139481

申请日：2023-04-26

Applicant: TOOLGEN INCORPORATED

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

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

CPC classification number: C12N15/907 ,  C12N9/22 ,  C12N9/644 ,  C12N15/86 ,  C12N15/11 ,  C12N2800/80 ,  C12N2310/20 ,  C12N2750/14143

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.

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.

公开(公告)号：US20190276503A1

公开(公告)日：2019-09-12

申请号：US16349672

申请日：2017-09-28

Applicant: TOOLGEN INCORPORATED

Inventor： Seok Joong KIM ,  Dong Woo SONG ,  Young Bin HONG ,  Byung Ok CHOI ,  Jae Young LEE ,  Jung Min LEE

IPC: C07K14/315 ,  C12N15/86 ,  A61K35/30

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.

公开(公告)号：US20230295652A1

公开(公告)日：2023-09-21

申请号：US18147156

申请日：2022-12-28

Applicant: TOOLGEN INCORPORATED

Inventor： Seokjoong KIM ,  Dong Woo SONG ,  Jae Young LEE ,  Jung Min LEE ,  Gyu-bon CHO ,  Hee Sook BAE

IPC: C12N15/85 ,  C12N15/113 ,  C12N9/22

CPC classification number: C12N15/85 ,  C12N9/22 ,  C12N15/113 ,  C12N2310/20

Abstract: The present invention relates to an expression control composition for controlling the expression of a duplicate gene or a method using the same. In addition, the present invention relates to a method of treating or improving a disease caused by gene duplication using the expression control composition for controlling the expression of a duplicate gene.