血脂异常：ANGPTL3 的体内基因组编辑：动脉粥样硬化的一种治疗方法？
Dyslipidaemia: In vivo genome editing of ANGPTL3: a therapy for atherosclerosis?
机构信息
Stanford Cardiovascular Institute, Stanford, CA, USA.
Department of Medicine, Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, CA, USA.
出版信息
Nat Rev Cardiol. 2018 May;15(5):259-260. doi: 10.1038/nrcardio.2018.38. Epub 2018 Apr 5.
Abstract
Hyperlipidemia is an important risk factor for coronary heart disease. Chadwick and colleagues report significantly reduced blood lipid levels following CRISPR-based genome editing in mice to introduce loss-of-function mutations in ANGPTL3, a lipoprotein lipase inhibitor. The treatments were effective in both healthy and Lplr−/− mice and comparable to PCSK9-targeted genome editing, without causing off-target mutations.
摘要
高脂血症是冠心病的一个重要危险因素。Chadwick 及其同事报道，通过 CRISPR 为基础的基因编辑使小鼠体内 ANGPTL3 产生失活突变，从而抑制脂蛋白脂肪酶，可显著降低血脂水平。这些治疗方法在健康和 Lplr−/−小鼠中均有效，且与 PCSK9 靶向基因编辑相当，而不会引起脱靶突变。
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