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利用无义介导的mRNA衰变规则进行基因组工程和个性化医疗。

Leveraging Rules of Nonsense-Mediated mRNA Decay for Genome Engineering and Personalized Medicine.

作者信息

Popp Maximilian W, Maquat Lynne E

机构信息

Department of Biochemistry and Biophysics, School of Medicine and Dentistry, University of Rochester, Rochester, NY 14642, USA; Center for RNA Biology, University of Rochester, Rochester, NY 14642, USA.

Department of Biochemistry and Biophysics, School of Medicine and Dentistry, University of Rochester, Rochester, NY 14642, USA; Center for RNA Biology, University of Rochester, Rochester, NY 14642, USA.

出版信息

Cell. 2016 Jun 2;165(6):1319-1322. doi: 10.1016/j.cell.2016.05.053.

Abstract

Nonsense-mediated mRNA decay (NMD) is a eukaryotic mRNA quality control and regulatory process that plays direct roles in human health and disease. In this Minireview, we discuss how understanding the molecular events that trigger NMD can facilitate strategic targeting of genes via CRISPR/Cas9 technologies and also inform disease diagnostics and treatments.

摘要

无义介导的mRNA降解(NMD)是一种真核生物mRNA质量控制和调节过程,在人类健康和疾病中发挥着直接作用。在这篇迷你综述中,我们讨论了理解触发NMD的分子事件如何能够通过CRISPR/Cas9技术促进对基因的靶向调控,同时也为疾病的诊断和治疗提供信息。

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