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.