COMPOSITIONS AND METHODS FOR THE MODIFICATION AND REGULATION OF LIVER GENE EXPRESSION
개요
발명자
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 분류
CPC 분류
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).
원문 (중국어)
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).