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公开(公告)号:US20230340435A1
公开(公告)日:2023-10-26
申请号:US17918092
申请日:2021-04-09
发明人: Alexandra Chadwick , Kallanthottathil G. Rajeev , Ellen Rohde , Christopher Cheng , Caroline Reiss
IPC分类号: C12N9/22 , C12N15/11 , C12N15/90 , A61K38/46 , A61K31/7105
CPC分类号: C12N9/22 , A61K31/7105 , A61K38/465 , C12N15/11 , C12N15/907 , C07K2319/09 , C07K2319/80 , C12N2310/20 , C12N2310/321
摘要: Provided herein are compositions for gene modification or editing and methods of using same to treat or prevent certain conditions. Specific compositions and methods capable of safely and effectively editing gene targets expressed in the liver to durably lower LDL-C thereby treating a leading cause of cardiovascular disease are disclosed.