CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia.
CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia.
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DOI：
10.1161/atvbaha.117.309326
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发表时间：
2018-01
期刊：
影响因子：
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通讯作者：
Musunuru K
中科院分区：
文献类型：
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作者：
Chadwick AC;Musunuru K
尽管人类遗传学已经鉴定出可以靶向预防动脉粥样硬化血管疾病的新型脂质相关基因，但靶向这些基因或其蛋白质产物的药物具有短期效应，并且需要在一生中频繁给药以获得最大益处。基因组编辑技术，如CRISPR-Cas9（成簇的规则间隔短回文重复序列-CRISPR相关9）有可能永久改变体内的基因，并产生长期甚至终身的抗动脉粥样硬化保护。在这篇综述中，我们讨论了基因组编辑技术的最新进展和小鼠体内体细胞基因组编辑的早期概念验证研究，这些研究强调了基因组编辑靶向患者疾病相关基因的潜力，这将为动脉粥样硬化建立一种新的治疗模式。
Although human genetics has resulted in the identification of novel lipid-related genes that can be targeted for the prevention of atherosclerotic vascular disease, medications targeting these genes or their protein products have short-term effects and require frequent administration during the course of the lifetime for maximal benefit. Genome-editing technologies, such as CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR–associated 9) have the potential to permanently alter genes in the body and produce long-term and even lifelong protection against atherosclerosis. In this review, we discuss recent advances in genome-editing technologies and early proof-of-concept studies of somatic in vivo genome editing in mice that highlight the potential of genome editing to target disease-related genes in patients, which would establish a novel therapeutic paradigm for atherosclerosis.