Strategies for the CRISPR-Based Therapeutics

Bin Li; Yuyu Niu; Weizhi Ji; Yizhou Dong

doi:10.1016/j.tips.2019.11.006

Strategies for the CRISPR-Based Therapeutics

Trends Pharmacol Sci. 2020 Jan;41(1):55-65. doi: 10.1016/j.tips.2019.11.006. Epub 2019 Dec 17.

Authors

Bin Li¹, Yuyu Niu², Weizhi Ji³, Yizhou Dong⁴

Affiliations

¹ Department of Infectious Disease, Shenzhen People's Hospital, The Second Clinical Medical College of Jinan University, The First Affiliated Hospital of Southern University of Science and Technology, Shenzhen 518020, China. Electronic address: libszph@yeah.net.
² Yunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, Yunnan 650500, China. Electronic address: niuyy@lpbr.cn.
³ Yunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming, Yunnan 650500, China. Electronic address: wji@lpbr.cn.
⁴ Division of Pharmaceutics and Pharmacology, College of Pharmacy, The Ohio State University, Columbus, Ohio 43210, USA. Electronic address: dong.525@osu.edu.

Abstract

The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many approaches to improve the genome editing activity of the CRISPR-Cas system and deliver its components both ex vivo and in vivo. Moreover, these strategies have been applied to genome editing in preclinical research and clinical trials. In this review, we focus on representative strategies for regulation and delivery of the CRISPR-Cas system, and outline current therapeutic applications in their clinical translation.

Keywords: CRISPR; CRISPR delivery; CRISPR-based therapeutics; gene editing.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Review

MeSH terms

Animals
CRISPR-Cas Systems*
Clustered Regularly Interspaced Short Palindromic Repeats*
Gene Editing / methods*
Humans

Grants and funding

R35 GM119679/GM/NIGMS NIH HHS/United States