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

Walker, HE, Rizzo, M, Fras, Z, Jug, B, Banach, M and Penson, PE (2021) CRISPR Gene Editing in Lipid Disorders and Atherosclerosis: Mechanisms and Opportunities. Metabolites, 11 (12). ISSN 2218-1989

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Open Access URL: https://doi.org/10.3390/metabo11120857 (Published version)


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: 0301 Analytical Chemistry, 0601 Biochemistry and Cell Biology, 1103 Clinical Sciences
Subjects: Q Science > QH Natural history > QH301 Biology
Q Science > QH Natural history > QH426 Genetics
R Medicine > RM Therapeutics. Pharmacology
Divisions: Pharmacy & Biomolecular Sciences
Publisher: MDPI AG
Date Deposited: 10 Dec 2021 10:23
Last Modified: 10 Dec 2021 10:30
DOI or ID number: 10.3390/metabo11120857
URI: https://researchonline.ljmu.ac.uk/id/eprint/15913
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