Leveraging Rules of Nonsense-Mediated mRNA Decay for Genome Engineering and Personalized Medicine.
Leveraging Rules of Nonsense-Mediated mRNA Decay for Genome Engineering and Personalized Medicine.
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DOI：
10.1016/j.cell.2016.05.053
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发表时间：
2016-06-02
期刊：
影响因子：
64.5
通讯作者：
Maquat LE
中科院分区：
文献类型：
--
作者：
Popp MW;Maquat LE
无义介导的mRNA衰变（NMD）是真核生物mRNA质量控制和调节过程，在人类健康和疾病中发挥直接作用。在这篇小综述中，我们讨论了如何理解触发NMD的分子事件，通过CRISPR/Cas9技术促进基因的战略靶向，并为疾病诊断和治疗提供信息。
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.