Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications

Yamin Li; Zachary Glass; Mingqian Huang; Zheng-Yi Chen; Qiaobing Xu

doi:10.1016/j.biomaterials.2019.119711

Ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications

Biomaterials. 2020 Mar:234:119711. doi: 10.1016/j.biomaterials.2019.119711. Epub 2020 Jan 10.

Authors

Yamin Li¹, Zachary Glass¹, Mingqian Huang², Zheng-Yi Chen³, Qiaobing Xu⁴

Affiliations

¹ Department of Biomedical Engineering, Tufts University, Medford, MA, 02155, USA.
² Eaton-Peabody Laboratory, Massachusetts Eye and Ear, Department of Otolaryngology-Head and Neck Surgery, Harvard Medical School, Boston, MA, 02114, USA.
³ Eaton-Peabody Laboratory, Massachusetts Eye and Ear, Department of Otolaryngology-Head and Neck Surgery, Harvard Medical School, Boston, MA, 02114, USA. Electronic address: Zheng-Yi_Chen@meei.harvard.edu.
⁴ Department of Biomedical Engineering, Tufts University, Medford, MA, 02155, USA. Electronic address: Qiaobing.Xu@tufts.edu.

Abstract

The recently developed CRISPR/Cas9 technology has revolutionized the genome engineering field. Since 2016, increasing number of studies regarding CRISPR therapeutics have entered clinical trials, most of which are focusing on the ex vivo genome editing. In this review, we highlight the ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. In these studies, CRISPR/Cas9 tools were used to edit cells in vitro and the successfully edited cells were considered as therapeutics, which can be introduced into patients to treat diseases. Considering a large number of previous reviews have been focused on the CRISPR/Cas9 delivery methods and materials, this review provides a different perspective, by mainly introducing the targeted conditions and design strategies for ex vivo CRISPR/Cas9 therapeutics. Brief descriptions of the history, functionality, and applications of CRISPR/Cas9 systems will be introduced first, followed by the design strategies and most significant results from previous research that used ex vivo CRISPR/Cas9 genome editing for the treatment of conditions or diseases. The last part of this review includes general information about the status of CRISPR/Cas9 therapeutics in clinical trials. We also discuss some of the challenges as well as the opportunities in this research area.

Keywords: CRISPR/Cas9; Clinical trials; Ex vivo; Genetic disease; Genome editing.

Publication types

Research Support, N.I.H., Extramural
Review

MeSH terms

CRISPR-Cas Systems / genetics
Clustered Regularly Interspaced Short Palindromic Repeats*
Gene Editing*
Genetic Therapy
Genome
Humans

Abstract

Publication types

MeSH terms

Grants and funding