In vivo genome editing of ANGPTL3: a therapy for atherosclerosis?

被引:10
|
作者
Rhee, June-Wha [1 ,2 ]
Wu, Joseph C. [1 ,2 ,3 ]
机构
[1] Stanford Cardiovasc Inst, Stanford, CA 94305 USA
[2] Stanford Univ, Sch Med, Dept Med, Div Cardiovasc Med, Stanford, CA 94305 USA
[3] Stanford Univ, Sch Med, Dept Radiol, Stanford, CA 94305 USA
关键词
DISEASE; DNA;
D O I
10.1038/nrcardio.2018.38
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Hyperlipidaemia is an important risk factor for coronary artery disease. Chadwick and colleagues report significantly reduced blood lipid levels following CRISPR-based in vivo genome editing in mice to introduce loss-of-function mutations in Angptl3, encoding a lipoprotein lipase inhibitor. Treatment was effective in both wild-type and Ldlr(-/-) mice and had a similar effect to that of Pcsk9-targeted genome editing, without causing off-target mutations.
引用
收藏
页码:259 / 260
页数:2
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