Moreno Ana M, Mali Prashant
Department of Bioengineering, University of California San Diego, San Diego, CA, USA.
Wiley Interdiscip Rev Syst Biol Med. 2017 Jul;9(4). doi: 10.1002/wsbm.1380. Epub 2017 Feb 15.
Differences in genomes underlie most organismal diversity, and aberrations in genomes underlie many disease states. With the growing knowledge of the genetic and pathogenic basis of human disease, development of safe and efficient platforms for genome and epigenome engineering will transform our ability to therapeutically target human diseases and also potentially engineer disease resistance. In this regard, the recent advent of clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated (Cas) RNA-guided nuclease systems have transformed our ability to target nucleic acids. Here we review therapeutic genome engineering applications with a specific focus on the CRISPR-Cas toolsets. We summarize past and current work, and also outline key challenges and future directions. WIREs Syst Biol Med 2017, 9:e1380. doi: 10.1002/wsbm.1380 For further resources related to this article, please visit the WIREs website.
基因组差异是大多数生物多样性的基础，而基因组畸变是许多疾病状态的基础。随着对人类疾病遗传和致病基础的了解不断增加，开发安全有效的基因组和表观基因组工程平台将改变我们治疗人类疾病的能力，并有可能设计出抗病能力。在这方面，成簇规律间隔短回文重复序列（CRISPR）-CRISPR相关（Cas）RNA引导核酸酶系统的最新出现改变了我们靶向核酸的能力。在此，我们回顾治疗性基因组工程应用，特别关注CRISPR-Cas工具集。我们总结了过去和当前的工作，并概述了关键挑战和未来方向。WIREs系统生物学与医学2017年，9：e1380。doi:10.1002/wsbm.1380 有关本文的更多资源，请访问WIREs网站。
