Department of Microbiology, Synthetic Biology Laboratory, School of Biological Sciences and Biotechnology, Institute of Advanced Research, Koba Institutional Area, Gandhinagar 382007, India.
Department of R&D, Cementic S.A.S., Paris 75000, France.
J Cell Biochem. 2018 Jan;119(1):81-94. doi: 10.1002/jcb.26165. Epub 2017 Jun 19.
The Type II CRISPR-Cas9 system is a simple, efficient, and versatile tool for targeted genome editing in a wide range of organisms and cell types. It continues to gain more scientific interest and has established itself as an extremely powerful technology within our synthetic biology toolkit. It works upon a targeted site and generates a double strand breaks that become repaired by either the NHEJ or the HDR pathway, modifying or permanently replacing the genomic target sequences of interest. These can include viral targets, single-mutation genetic diseases, and multiple-site corrections for wide scale disease states, offering the potential to manage and cure some of mankind's most persistent biomedical menaces. Here, we present the developing progress and future potential of CRISPR-Cas9 in biological and biomedical investigations, toward numerous therapeutic, biomedical, and biotechnological applications, as well as some of the challenges within. J. Cell. Biochem. 119: 81-94, 2018. © 2017 Wiley Periodicals, Inc.
II 型 CRISPR-Cas9 系统是一种简单、高效、多功能的靶向基因组编辑工具，可用于广泛的生物和细胞类型。它继续引起更多的科学兴趣，并在我们的合成生物学工具包中确立了自己作为一种极其强大的技术。它在靶向位点发挥作用，产生双链断裂，这些断裂可以通过 NHEJ 或 HDR 途径修复，从而修饰或永久替换感兴趣的基因组靶序列。这些可以包括病毒靶标、单突变遗传疾病以及大规模疾病状态的多靶点校正，从而有可能管理和治疗人类一些最顽固的生物医学威胁。在这里，我们介绍了 CRISPR-Cas9 在生物学和生物医学研究中的发展进展和未来潜力，以及在治疗、生物医学和生物技术应用方面的一些挑战。J. Cell. Biochem. 119: 81-94, 2018. © 2017 Wiley Periodicals, Inc.
