公开(公告)号：US11845951B2

公开(公告)日：2023-12-19

申请号：US16650993

申请日：2018-09-28

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

发明人： Dong Woo Song ,  Jung Min Lee ,  Un Gi Kim ,  Jeong Hun Kim ,  Dong Hyun Jo

IPC分类号： C12N15/86 ,  C12N15/113 ,  C12N9/22 ,  A61P27/02 ,  A61K48/00

CPC分类号： C12N15/86 ,  A61P27/02 ,  C12N9/22 ,  C12N15/113 ,  A61K48/00 ,  C12N2310/20 ,  C12N2800/80

摘要： 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：US11999952B2

公开(公告)日：2024-06-04

申请号：US17236660

申请日：2021-04-21

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

发明人： Jeong Hun Kim ,  Sung Wook Park ,  Seokjoong Kim ,  Dong Woo Song

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

CPC分类号： C12N15/11 ,  A61K9/0048 ,  A61K38/465 ,  A61K48/00 ,  A61K48/005 ,  C07K14/4702 ,  C07K14/515 ,  C07K14/52 ,  C12N9/22 ,  C12N15/113 ,  C12N15/1136 ,  C12N15/86 ,  A01K2227/105 ,  A01K2267/03 ,  C12N2310/20 ,  C12N2750/14143 ,  Y02A50/30

摘要： 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：US11667934B2

公开(公告)日：2023-06-06

申请号：US16623017

申请日：2018-06-15

申请人： TOOLGEN INCORPORATED

发明人： Seokjoong Kim ,  Dong Woo Song ,  Kyu Jun Lee ,  Jung-Min Lee ,  Un-Gi Kim

IPC分类号： C12N15/90 ,  C12N9/22 ,  C12N9/64 ,  C12N15/11 ,  C12N15/86 ,  A61K38/17 ,  A61P7/04 ,  A61K48/00 ,  C07K14/775 ,  C12N15/113

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

摘要： 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：US11572574B2

公开(公告)日：2023-02-07

申请号：US16776707

申请日：2020-01-30

申请人： TOOLGEN INCORPORATED

发明人： Seokjoong Kim ,  Dong Woo Song ,  Jae Young Lee ,  Jung Min Lee ,  Gyu-bon Cho ,  Hee Sook Bae

IPC分类号： C07H21/02 ,  C07H21/04 ,  C12N15/85 ,  C12N15/113 ,  C12N9/22

摘要： 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.