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Review
. 2023 Jun;43(6):832-835.
doi: 10.1161/ATVBAHA.122.318354. Epub 2023 Apr 27.

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

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Free article
Review

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

Menno Hoekstra et al. Arterioscler Thromb Vasc Biol. 2023 Jun.
Free article

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.

Keywords: cardiovascular disease; editing; hypercholesterolemic; hyperlipidemia; therapeutic effect.

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