Gene editing of PCSK9 or ANGPTL3 and compositions and methods of using same for treatment of disease

Abstract

A gene editing system comprising: (a) a gene editor fusion protein comprising a Cas protein and a deaminase, or a nucleic acid encoding the gene editor fusion protein, and (b) a hybrid guide nucleic acid comprising a spacer sequence, wherein the spacer sequence comprises at least one deoxyribonucleotide and a plurality of ribonucleotide is claimed. A 2’ hydroxyl group of a ribose on at least one ribonucleotide may be covalently linked to a methyl group (2’-OMe). The gene editing system for use in treating or preventing an atherosclerotic cardiovascular disease, or lowering blood low-density lipoprotein cholesterol (LDL-C) levels or lowering blood triglyceride levels is further claimed. [None]