KIM, SEOKJOONG,SONG, DONG WOO,LEE, KYU JUN,LEE, JUNG-MIN,KIM, UN-GI
申请号：
CA3067316
公开号：
CA3067316A1
申请日:
2018.06.15
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
The present specification pertains to 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 disease or for improving a body function by inserting a transgene capable of normal functions and expression (e.g., therapeutic gene), instead of a diseased gene that functions or is expressed abnormally, into a high-expression secretory gene and inducing the expression of the transgene. The high-expression secretory gene includes an HP or APOC3 gene. The transgene includes what is expressed at a high level by means of a promoter in the hepatocyte genome and secreted extracellularly.
