COMPOSITIONS AND METHODS FOR THE MODIFICATION AND REGULATION OF LIVER GENE EXPRESSION
卷宗概要
发明人
Raymond D. HICKEY; Sean CHEN; Ymer Leif Michael BJORNSON; Benjamin Julius RAUCH; William Douglass WRIGHT; Stepan TYMOSHENKO; Wiputra Jaya HARTONO; Aaron DELOUGHERY; Lucas Benjamin HARRINGTON; Lauren Kelli UYESAKA
IPC 分类
CPC 分类
Provided herein are compositions, systems, and methods for modifying a human APOC3 gene, PCSK9 gene, or ANGPTL3 gene. Systems, compositions, and methods may comprise a CRISPR-associated (Cas) protein or uses thereof. Systems, compositions, and methods of the present disclosure may be useful for treatment of APOC3 associated conditions, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG).
原文（中文）
Provided herein are compositions, systems, and methods for modifying a human APOC3 gene, PCSK9 gene, or ANGPTL3 gene. Systems, compositions, and methods may comprise a CRISPR-associated (Cas) protein or uses thereof. Systems, compositions, and methods of the present disclosure may be useful for treatment of APOC3 associated conditions, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG).