使用CRISPR/Cas9基因组编辑技术治疗血脂异常
Treatment of Dyslipidemia Using CRISPR/Cas9 Genome Editing.
作者信息
Chadwick Alexandra C, Musunuru Kiran
机构信息
Cardiovascular Institute, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
Department of Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
出版信息
Curr Atheroscler Rep. 2017 Jul;19(7):32. doi: 10.1007/s11883-017-0668-8.
PURPOSE OF REVIEW
Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated 9 (Cas9) has recently emerged as a top genome editing technology and has afforded investigators the ability to more easily study a number of diseases. This review discusses CRISPR/Cas9's advantages and limitations and highlights a few recent reports on genome editing applications for alleviating dyslipidemia through disruption of proprotein convertase subtilisin/kexin type 9 (PCSK9).
RECENT FINDINGS
Targeting of mouse Pcsk9 using CRISPR/Cas9 technology has yielded promising results for lowering total cholesterol levels, and several recent findings are highlighted in this review. Reported on-target mutagenesis efficiency is as high as 90% with a subsequent 40% reduction of blood cholesterol levels in mice, highlighting the potential for use as a therapeutic in human patients. The ability to characterize and treat diseases is becoming easier with the recent advances in genome editing technologies. In this review, we discuss how genome editing strategies can be of use for potential therapeutic applications.
综述目的
成簇规律间隔短回文重复序列（CRISPR）/CRISPR相关蛋白9（Cas9）最近已成为一种顶级基因组编辑技术，使研究人员能够更轻松地研究多种疾病。本综述讨论了CRISPR/Cas9的优势和局限性，并重点介绍了一些最近关于通过破坏前蛋白转化酶枯草溶菌素/kexin 9型（PCSK9）来缓解血脂异常的基因组编辑应用报告。
最新发现
使用CRISPR/Cas9技术靶向小鼠Pcsk9在降低总胆固醇水平方面已取得了有前景的结果，本综述重点介绍了一些最近的发现。报道的靶向诱变效率高达90%，随后小鼠血液胆固醇水平降低40%，凸显了其在人类患者中用作治疗方法的潜力。随着基因组编辑技术的最新进展，表征和治疗疾病变得更加容易。在本综述中，我们讨论了基因组编辑策略如何可用于潜在的治疗应用。
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