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本文引用的文献

1
A novel humanized mouse lacking murine P450 oxidoreductase for studying human drug metabolism.一种用于研究人类药物代谢的新型缺失小鼠P450氧化还原酶的人源化小鼠。
Nat Commun. 2017 Jun 28;8(1):39. doi: 10.1038/s41467-017-00049-x.
2
Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease.利用 CRISPR/Cas9 进行体细胞基因组编辑可产生并纠正代谢疾病。
Sci Rep. 2017 Mar 16;7:44624. doi: 10.1038/srep44624.
3
CRISPR-Cas9 cleavage efficiency correlates strongly with target-sgRNA folding stability: from physical mechanism to off-target assessment.CRISPR-Cas9 切割效率与靶 sgRNA 折叠稳定性密切相关：从物理机制到脱靶评估。
Sci Rep. 2017 Mar 10;7(1):143. doi: 10.1038/s41598-017-00180-1.
4
CRISPR knockout rat cytochrome P450 3A1/2 model for advancing drug metabolism and pharmacokinetics research.CRISPR 基因敲除大鼠细胞色素 P4503A1/2 模型用于推进药物代谢和药代动力学研究。
Sci Rep. 2017 Feb 20;7:42922. doi: 10.1038/srep42922.
5
A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo.一种用于在体内治疗性靶向乙肝病毒（HBV）DNA和前蛋白转化酶枯草溶菌素9（pcsk9）的非病毒CRISPR/Cas9递送系统。
Cell Res. 2017 Mar;27(3):440-443. doi: 10.1038/cr.2017.16. Epub 2017 Jan 24.
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Eukaryotic elongation factor 2 is a prognostic marker and its kinase a potential therapeutic target in HCC.真核生物延伸因子2是一种预后标志物，其激酶是肝癌潜在的治疗靶点。
Oncotarget. 2017 Feb 14;8(7):11950-11962. doi: 10.18632/oncotarget.14447.
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IDH1 deficiency attenuates gluconeogenesis in mouse liver by impairing amino acid utilization.异柠檬酸脱氢酶1（IDH1）缺乏通过损害氨基酸利用来减弱小鼠肝脏中的糖异生作用。
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New CRISPR-Cas systems from uncultivated microbes.来自未培养微生物的新型CRISPR-Cas系统。
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CRISPR/Cas9：处于肝病学前沿

CRISPR/Cas9: at the cutting edge of hepatology.

作者信息

Pankowicz Francis P, Jarrett Kelsey E, Lagor William R, Bissig Karl-Dimiter

机构信息

Center for Cell and Gene Therapy, Center for Stem Cells and Regenerative Medicine, Baylor College of Medicine, Houston, Texas, USA.

Graduate Program Department of Molecular & Cellular Biology, Baylor College of Medicine, Houston, Texas, USA.

出版信息

Gut. 2017 Jul;66(7):1329-1340. doi: 10.1136/gutjnl-2016-313565. Epub 2017 May 9.

DOI:10.1136/gutjnl-2016-313565
PMID:28487442
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5878048/
Abstract

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 genome engineering has revolutionised biomedical science and we are standing on the cusp of medical transformation. The therapeutic potential of this technology is tremendous, however, its translation to the clinic will be challenging. In this article, we review recent progress using this genome editing technology and explore its potential uses in studying and treating diseases of the liver. We discuss the development of new research tools and animal models as well as potential clinical applications, strategies and challenges.

摘要

成簇规律间隔短回文重复序列（CRISPR）/Cas9基因组工程已经彻底改变了生物医学科学，我们正站在医学变革的风口浪尖。这项技术的治疗潜力巨大，然而，将其转化应用于临床将具有挑战性。在本文中，我们回顾了使用这种基因组编辑技术的最新进展，并探讨了其在研究和治疗肝脏疾病方面的潜在用途。我们讨论了新研究工具和动物模型的开发以及潜在的临床应用、策略和挑战。