Bhushan Kul, Chattopadhyay Anirudha, Pratap Dharmendra
Advanced Centre for Plant Virology, Division of Plant Pathology, Indian Agricultural Research Institute, New Delhi, India.
Department of Plant Pathology, C.P. College of Agriculture, S.D. Agricultural University, S.K. Nagar, Gujrat, India.
Biotechnol Lett. 2018 Mar;40(3):465-477. doi: 10.1007/s10529-018-2506-7. Epub 2018 Jan 17.
Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system allows biologists to edit genomic DNA of any cell in precise and specific way, entailing great potential for crop improvement, drug development and gene therapy. The system involves a nuclease (Cas9) and a designed guide RNA that are involved in wide range of applications such as genome modification, transcriptional modulation, genomic loci marking and RNA tracking. The limitation of the technique, in view of resistance of thymidine-rich genome to Cas9 cleavage, has now been overcome by the use of Cpf1 nuclease. In this review, we present an overview of CRISPR nucleases (Cas9 or Cpf1) with particular emphasis on human genome modification and compare their advantages and limitations. Furthermore, we summarize some of the pros and cons of CRISPR technology particularly in human therapeutics.
成簇规律间隔短回文重复序列（CRISPR）/Cas9系统使生物学家能够以精确且特定的方式编辑任何细胞的基因组DNA，在作物改良、药物开发和基因治疗方面具有巨大潜力。该系统包含一种核酸酶（Cas9）和一条设计好的引导RNA，它们参与了多种应用，如基因组修饰、转录调控、基因组位点标记和RNA追踪。鉴于富含胸腺嘧啶的基因组对Cas9切割具有抗性，该技术的局限性现已通过使用Cpf1核酸酶得以克服。在本综述中，我们概述了CRISPR核酸酶（Cas9或Cpf1），特别强调了对人类基因组的修饰，并比较了它们的优缺点。此外，我们总结了CRISPR技术的一些利弊，特别是在人类治疗方面。
