Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research

Liting You; Ruizhan Tong; Mengqian Li; Yuncong Liu; Jianxin Xue; You Lu

doi:10.1016/j.omtm.2019.02.008

Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research

Mol Ther Methods Clin Dev. 2019 Mar 15:13:359-370. doi: 10.1016/j.omtm.2019.02.008. eCollection 2019 Jun 14.

Authors

Liting You¹, Ruizhan Tong¹, Mengqian Li¹, Yuncong Liu^{1

2}, Jianxin Xue¹, You Lu¹

Affiliations

¹ Department of Thoracic Cancer, Cancer Center, West China Hospital, West China School of Medicine, Sichuan University, 37 Guoxue Lane, Chengdu, Sichuan 610041, China.
² Department of Gynaecological Oncology, Guizhou Provincial People's Hospital, 83 Zhongshan Dong Road, Guiyang, Guizhou 550002, China.

Abstract

The expanding CRISPR-Cas9 technology is an easily accessible, programmable, and precise gene-editing tool with numerous applications, most notably in biomedical research. Together with advancements in genome and transcriptome sequencing in the era of metadata, genomic engineering with CRISPR-Cas9 meets the developmental requirements of precision medicine, and clinical tests using CRISPR-Cas9 are now possible. This review summarizes developments and established preclinical applications of CRISPR-Cas9 technology, along with its current challenges, and highlights future applications in translational research.

Keywords: CRISPR-Cas9; gene editing; gene therapy; off-target; tumor immunotherapy.

Publication types

Review