The application of genome editing technology

Jianwei Zhu, Wenjing Ma, Ziwei Huang, Qiuyu Zhang, Xiaoying Xie, Xiaoming Yang, Hualin Sun


Genome editing technology is currently the most effective tool for accurately manipulating genomes at specific locations. Zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered regularly interspaced short palindromic repeats associated Cas9 (CRISPR/Cas9) system were used genome editing technologies. Both editing done with ZFNs and TALENs consist of DNA-binding domains which are fused to endonucleases. This is done to enable a broad range of genetic modifications by inducing DNA double-strand breaks (DSB) that stimulate the error-prone Non-Homologous End-Joining (NHEJ) or a homology-directed repair (HDR) at specific genomic locations. Different to ZFNs and TALENs, the CRISPR/Cas system is an RNA-mediated specific recognition process which DSB been introduced after the sgRNA binds to the targeted DNA sequence. The ZFNs, TALENs and CRISPR/Cas9 systems modify these genomic characteristics precisely through these repair mechanisms, and have been successfully used to manipulate the genome in human cells. These genome editing tools can be used to investigate gene function, to explore the genetic mechanisms of a disease, to discover new therapeutic targets, and to develop new disease models. Moreover, these genome editing technologies have a great potential in gene therapy. Here, we review the latest advances in the application of genome editing technology for the study and treatment of genetic diseases [Duchenne muscular dystrophy (DMD), hemophilia], cancers [chimeric antigen receptor T-cell immunotherapy (CAR-T) technology], viral infections [human immunodeficiency virus (HIV), hepatitis B virus (HBV)], bio-agricultures, and microorganisms.