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Engineered meganucleases specific for recognition sequences in the PCSK9 gene
专利权人:
Precision BioSciences; Inc.
发明人:
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.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/
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