CRISPR Gene Editing in Lipid Disorders and Atherosclerosis: Mechanisms and Opportunities

Walker, Harry E, Rizzo, Manfredi, Fras, Zlatko, Jug, Borut, Banach, Maciej and Penson, Peter E ORCID: 0000-0001-6763-1489 (2021) CRISPR Gene Editing in Lipid Disorders and Atherosclerosis: Mechanisms and Opportunities. METABOLITES, 11 (12). 857-.

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Official URL: http://dx.doi.org/10.3390/metabo11120857

Abstract

Elevated circulating concentrations of low-density lipoprotein cholesterol (LDL-C) have been conclusively demonstrated in epidemiological and intervention studies to be causally associated with the development of atherosclerotic cardiovascular disease. Enormous advances in LDL-C reduction have been achieved through the use of statins, and in recent years, through drugs targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), a key regulator of the hepatic LDL-receptor. Existing approaches to PCSK9 targeting have used monoclonal antibodies or RNA interference. Although these approaches do not require daily dosing, as statins do, repeated subcutaneous injections are nevertheless necessary to maintain effectiveness over time. Recent experimental studies suggest that clustered regularly interspaced short palindromic repeats (CRISPR) gene-editing targeted at PCSK9 may represent a promising tool to achieve the elusive goal of a 'fire and forget' lifelong approach to LDL-C reduction. This paper will provide an overview of CRISPR technology, with a particular focus on recent studies with relevance to its potential use in atherosclerotic cardiovascular disease.

Item Type:	Article
Uncontrolled Keywords:	CRISPR, atherosclerosis, dyslipidaemia
Divisions:	Faculty of Health and Life Sciences Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences
Depositing User:	Symplectic Admin
Date Deposited:	06 May 2022 15:27
Last Modified:	18 Jan 2023 21:04
DOI:	10.3390/metabo11120857
Open Access URL:	https://www.mdpi.com/2218-1989/11/12/857
Related URLs:	Author Publisher
URI:	https://livrepository.liverpool.ac.uk/id/eprint/3154394