Advances in the application strategies of CRISPR/Cas9 technology in chimeric antigen receptor T cell therapy for hematological malignancies
10.3760/cma.j.cn121090-20240911-00343
- VernacularTitle:CRISPR/Cas9技术在血液肿瘤CAR-T细胞疗法中应用策略的研究进展
- Author:
Yuwen WANG
1
;
Yongmin TANG
1
Author Information
1. 浙江大学医学院附属儿童医院血液肿瘤中心,浙江省儿童白血病诊治技术研究中心,国家儿童健康与疾病临床医学研究中心,杭州 310003
- Publication Type:Journal Article
- From:
Chinese Journal of Hematology
2025;46(5):481-488
- CountryChina
- Language:Chinese
-
Abstract:
Chimeric antigen receptor (CAR) T-cell therapy has achieved breakthroughs in treating relapsed/refractory B-cell malignancies. However, it still faces challenges, including complex manufacturing processes, limited indications, T-cell exhaustion, and insufficient durability of therapeutic efficacy. CRISPR/Cas9, a highly efficient and relatively simple gene-editing technology, offers new avenues for overcoming these limitations. This review briefly outlines the working mechanism of CRISPR/Cas9 and focuses on its recent applications and clinical practices in developing universal CAR T-cells, enhancing T-cell function, and extending CAR T-cell therapy to T-cell and myeloid leukemias. Furthermore, this review highlights optimization strategies developed over the past two years to enhance the editing precision, delivery efficiency, and safety of the CRISPR/Cas9 system, aiming to provide insights for the optimal design and clinical application of CAR T-cell therapy.