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

Current advances of CRISPR-Cas technology in cell therapy

Hou-Yuan Qiu  1 , Rui-Jin Ji  1 , Ying Zhang  1
Affiliations
Review

Current advances of CRISPR-Cas technology in cell therapy

Hou-Yuan Qiu et al. Cell Insight. .

Erratum in

  • Corrigendum to previous published articles.
    [No authors listed] [No authors listed] Cell Insight. 2025 Jan 11;4(2):100225. doi: 10.1016/j.cellin.2024.100225. eCollection 2025 Apr. Cell Insight. 2025. PMID: 39881711 Free PMC article.

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.

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Conflict of interest statement

The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Figures

Fig. 1
Fig. 1
| CRISPR toolbox for translational study. Illustration of three generations of CRISPR-based genome editing tools: nucleases, base editors and prime editors. Examples of substrates and edited products are shown, along with CRISPR-Cas tools that can be used to achieve the target editing. PAM, protospacer adjacent motif; NHEJ, non-homologous end joining; HDR, homology-directed repair; UGI, uracil glycosylase inhibitor (UGI).
Fig. 2
Fig. 2
| The flow chart of CRISPR-based cell therapy. (Left) CRISPR-mediated cell therapies begin with the isolation of cells. Cells are cultured ex vivo and edited with CRISPR-tools before infusing back to patient. (Right) CRISPR-edited iPSCs therapy. iPSCs originally reprogramed from somatic cells can serve as a powerful cell source for various cell based therapies. CRISPR edited iPSCs are differentiated into cell types of interest, and differentiated cells are then transfused back into patient. iPSCs, induced pluripotent stem cells; NK cells, natural killer cells.
Fig. 3
Fig. 3
| Major landmarks in CRISPR/Cas development and their progress in cell therapy. Timeline highlighting the major developments in the fields of CRISPR-Cas based cell therapies, including the discovery of CRISPR toolbox and major clinical advances.
See this image and copyright information in PMC

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