Current advances of CRISPR-Cas technology in cell therapy

Hou-Yuan Qiu; Rui-Jin Ji; Ying Zhang

doi:10.1016/j.cellin.2022.100067

Current advances of CRISPR-Cas technology in cell therapy

Cell Insight. 2022 Oct 26;1(6):100067. doi: 10.1016/j.cellin.2022.100067. eCollection 2022 Dec.

Authors

Hou-Yuan Qiu¹, Rui-Jin Ji¹, Ying Zhang¹

Affiliation

¹ Department of Rheumatology and Immunology, Medical Research Institute, Frontier Science Center for Immunology and Metabolism, Zhongnan Hospital of Wuhan University, Wuhan University, Wuhan, 430071, China.

Abstract

CRISPR-Cas is a versatile genome editing technology that has been broadly applied in both basic research and translation medicine. Ever since its discovery, the bacterial derived endonucleases have been engineered to a collection of robust genome-editing tools for introducing frameshift mutations or base conversions at site-specific loci. Since the initiation of first-in-human trial in 2016, CRISPR-Cas has been tested in 57 cell therapy trials, 38 of which focusing on engineered CAR-T cells and TCR-T cells for cancer malignancies, 15 trials of engineered hematopoietic stem cells treating hemoglobinopathies, leukemia and AIDS, and 4 trials of engineered iPSCs for diabetes and cancer. Here, we aim to review the recent breakthroughs of CRISPR technology and highlight their applications in cell therapy.

Keywords: CRISPR; Cas9; Cell therapy; Genome editing; HSPC; T cell; iPSC.

Publication types

Review