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.
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。
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