发明公开
- 专利标题: BASE EDITING OF ANGPTL3 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE
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申请号: US17918092申请日: 2021-04-09
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公开(公告)号: US20230340435A1公开(公告)日: 2023-10-26
- 发明人: Alexandra Chadwick , Kallanthottathil G. Rajeev , Ellen Rohde , Christopher Cheng , Caroline Reiss
- 申请人: Verve Therapautics, Inc.
- 申请人地址: US MA Boston
- 专利权人: Verve Therapautics, Inc.
- 当前专利权人: Verve Therapautics, Inc.
- 当前专利权人地址: US MA Boston
- 国际申请: PCT/US2021/026729 2021.04.09
- 进入国家日期: 2022-10-10
- 主分类号: C12N9/22
- IPC分类号: C12N9/22 ; C12N15/11 ; C12N15/90 ; A61K38/46 ; A61K31/7105
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.
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