Department of Foundations of Medicine, NYU Long Island School of Medicine, Mineola, NY, 11501, USA.
Department of Biochemistry and Molecular Biology, Hospices Civils de Lyon, Lyon, France.
Curr Atheroscler Rep. 2023 May;25(5):209-217. doi: 10.1007/s11883-023-01095-1. Epub 2023 Mar 13.
This review is aimed at providing an overview of new developments in gene editing technology, including examples of how this technology has been used to develop cell models for studying the effects of gene ablation or missense mutations on lipoprotein assembly and secretion.
CRISPR/Cas9-mediated gene editing is superior to other technologies because of its ease, sensitivity, and low off-target effects. This technology has been used to study the importance of microsomal triglyceride transfer protein in the assembly and secretion of apolipoprotein B-containing lipoproteins, as well as to establish causal effects of APOB gene missense mutations on lipoprotein assembly and secretion. CRISPR/Cas9 technology is anticipated to provide unprecedented flexibility in studying protein structure and function in cells and animals and to yield mechanistic insights into variants in the human genome.
本文旨在概述基因编辑技术的新进展,包括该技术如何用于构建细胞模型,以研究基因缺失或错义突变对脂蛋白组装和分泌的影响。
CRISPR/Cas9 介导的基因编辑因其简便、灵敏和低脱靶效应而优于其他技术。该技术已被用于研究微粒体甘油三酯转移蛋白在载脂蛋白 B 脂蛋白组装和分泌中的重要性,以及确定 APOB 基因突变对脂蛋白组装和分泌的因果影响。CRISPR/Cas9 技术有望为在细胞和动物中研究蛋白质结构和功能提供前所未有的灵活性,并深入了解人类基因组中的变异。