Perspective: Hepatocyte-Directed Base Editing as Novel Treatment for Human Dyslipidemia—Current Status and Remaining Challenges

• Published in: Arteriosclerosis, thrombosis, and vascular biology Vol. 43; no. 6; pp. 832 - 835
• Main Authors: Hoekstra, Menno, Van Eck, Miranda, Van Berkel, Theo J.C.
• Format: Journal Article
• Language: English
• Published: United States Lippincott Williams & Wilkins 01.06.2023
• Subjects: Cardiovascular Diseases - drug therapy; Cardiovascular Diseases - therapy; Dyslipidemias - drug therapy; Dyslipidemias - genetics; Gene Editing; Hepatocytes; Humans; Hypolipidemic Agents - therapeutic use; hypercholesterolemic; cardiovascular disease; hyperlipidemia; therapeutic effect; editing
• ISSN: 1079-5642; 1524-4636; 1524-4636
• DOI: 10.1161/ATVBAHA.122.318354

Hyperlipidemia is a major risk factor for the development of atherosclerotic cardiovascular disease. Lipid-lowering drug therapies therefore still form the heart of the ongoing battle against the occurrence of cardiovascular events. However, in light of the important improvements in gene interference and editing that have been made during the last 2 decades, gene therapy—the genetic modification of cells to produce a permanent therapeutic effect—is currently employed to relief hypercholesterolemic subjects from their potential (chronic) cardiovascular disease burden. In this perspective, we review the current status regarding hepatocyte-directed base editing to treat human dyslipidemia and provide suggestions for further technological improvement.