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

Perspective: Hepatocyte-Directed Base Editing as Novel Treatment for Human Dyslipidemia—Current... 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. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Arteriosclerosis Thrombosis and Vascular Biology Wolters Kluwer Health

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

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References (16)

Publisher
Wolters Kluwer Health
Copyright
© 2023 American Heart Association, Inc.
ISSN
1079-5642
eISSN
1524-4636
DOI
10.1161/atvbaha.122.318354
Publisher site
See Article on Publisher Site

Abstract

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.

Journal

Arteriosclerosis Thrombosis and Vascular BiologyWolters Kluwer Health

Published: Jun 27, 2023

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