Bartsevich, Victor,Jantz, Derek,Smith, James Jefferson,Lape, Janel
申请号：
AU2018254576
公开号：
AU2018254576A1
申请日:
2018.04.20
申请国别(地区):
AU
年份:
2019
代理人:
摘要：
The present invention encompasses engineered meganucleases which recognize and cleave a recognition sequence within the human PCSK9 gene. The present invention also encompasses methods for using such engineered meganucleases in a pharmaceutical composition and in methods for treating or reducing the symptoms of cholesterol-related disorders, such as hypercholesterolemia. Further, the invention encompasses pharmaceutical compositions comprising engineered meganuclease proteins, nucleic acids encoding engineered meganucleases, and the use of such compositions for treating cholesterol-related disorders, such as hypercholesterolemia.
