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.