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
相关论文
共 50 条
  • [11] Circulating ANGPTL3 and ANGPTL4 levels predict coronary artery atherosclerosis severity
    Ting Sun
    Wanlin Zhan
    Lijiang Wei
    Zuojun Xu
    Li Fan
    Yang Zhuo
    Changqian Wang
    Junfeng Zhang
    Lipids in Health and Disease, 20
  • [12] Protein region important for regulation of lipid metabolism in angiopoietin-like 3 (ANGPTL3) -: ANGPTL3 is cleaved and activated in vivo
    Ono, M
    Shimizugawa, T
    Shimamura, M
    Yoshida, K
    Noji-Sakikawa, C
    Ando, Y
    Koishi, R
    Furukawa, H
    JOURNAL OF BIOLOGICAL CHEMISTRY, 2003, 278 (43) : 41804 - 41809
  • [13] Circulating ANGPTL3 and ANGPTL4 levels predict coronary artery atherosclerosis severity
    Sun, Ting
    Zhan, Wanlin
    Wei, Lijiang
    Xu, Zuojun
    Fan, Li
    Zhuo, Yang
    Wang, Changqian
    Zhang, Junfeng
    LIPIDS IN HEALTH AND DISEASE, 2021, 20 (01)
  • [14] Structure and Function of Angiopoietin-like Protein 3 (ANGPTL3) in Atherosclerosis
    Lu, Xinjie
    CURRENT MEDICINAL CHEMISTRY, 2020, 27 (31) : 5159 - 5174
  • [15] Oligonucleotides targeting ANGPTL3
    Luo, Fei
    Guo, Yuan
    Li, Xiangping
    Hu, Xinqun
    Fang, Zhenfei
    INTERNATIONAL JOURNAL OF CARDIOLOGY, 2018, 256 : 8 - 8
  • [16] AN INVESTIGATIONAL IN VIVO BASE EDITING MEDICINE TARGETING ANGPTL3, VERVE-201, ACHIEVES PRECISE AND DURABLE LIVER EDITING IN NONCLINICAL STUDIES
    Lee, Richard
    Mazzola, Anne
    Denizio, Jamie
    Mizoguchi, Taiji
    Clendaniel, Victoria
    Garrity, Ryan
    Cox, Nicholas
    Glass, Zachary
    Chamarthi, Hareesh
    Flannigan, Sydney
    Matsumoto, Yuri
    Braun, Maurine
    Kathiresan, Sekar
    Biedenkapp, Joseph
    Rohde, Ellen
    Bellinger, Andrew
    Khera, Amit
    ATHEROSCLEROSIS, 2024, 395
  • [17] SEQUENTIAL IN VIVO CRISPR BASE EDITING OF THE PCSK9 AND ANGPTL3 GENES IN NON-HUMAN PRIMATES
    Bellinger, Andrew
    Rhode, Ellen
    Kathiresan, Sekar
    JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2022, 79 (09) : 953 - 953
  • [18] ANGPTL3 inhibition for hypercholesterolaemia
    Lim G.B.
    Nature Reviews Cardiology, 2021, 18 (2) : 72 - 72
  • [19] ANGPTL3 as therapeutic target
    Kersten, Sander
    CURRENT OPINION IN LIPIDOLOGY, 2021, 32 (06) : 335 - 341
  • [20] Blocking ANGPTL3 and CD47 impact on atherosclerosis-correspondence
    Hou, Han-Yong
    Chen, Yan-Shiang
    Yong, Su-Boon
    Yii, Chin-Yuan
    Su, Yu-Tsun
    PHARMACOLOGICAL RESEARCH, 2025, 212