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In vivo gene editing in lipid and atherosclerosis research.pdf (1.12 MB)
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In Vivo Gene Editing in Lipid and Atherosclerosis Research

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journal contribution
posted on 2023-02-07, 18:10 authored by Marco De Giorgi, Kelsey Jarrett, Thomas Vallim, William R. Lagor

The low-density lipoprotein receptor (Ldlr) and apolipoprotein E (Apoe) germline knockout (KO) models

have provided fundamental insights in lipid and atherosclerosis research for decades. However, testing new

candidate genes in these models requires extensive breeding, which is highly time and resource consuming.

In this chapter, we provide methods for rapidly modeling hypercholesterolemia and atherosclerosis as well

as testing new genes in adult mice through somatic gene editing. Adeno-associated viral (AAV) vectors are

exploited to deliver the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9

genome editing system (AAV-CRISPR) to the liver. This tool enables rapid and efficient editing of lipidand

atherosclerosis-related genes in the liver.


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