KIM, Seokjoong,SONG, Dong Woo,LEE, Kyu Jun,LEE, Jung-Min,KIM, Un-Gi
申请号:
SG11201911623W
公开号:
SG11201911623WA
申请日:
2018.06.15
申请国别(地区):
SG
年份:
2020
代理人:
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.