№ US20260002151A1발명
심사 중COMPOSITIONS AND METHODS FOR THE MODIFICATION AND REGULATION OF LIVER GENE EXPRESSION
COMPOSITIONS AND METHODS FOR THE MODIFICATION AND REGULATION OF LIVER GENE EXPRESSION
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).
❖Mammoth Biosciences, Inc.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§ 3/35 청구항C12N 15/11, A61P 5/, C12N 9/22 +39