Gong Jingli, Soleimani Samarkhazan Hamed, Siavashi Mohammad, Servatian Nazli, Pirsavabi Farnaz
College of Pharmacy, Jilin Medical University, Jilin, 132013, Jilin Province, China.
Student Research Committee, Department of Hematology and Blood Banking, School of Allied Medical Sciences, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
Mol Biol Rep. 2025 Nov 15;53(1):90. doi: 10.1007/s11033-025-11235-2.
Together with base editors, prime editors, and alternative nuclease platforms, CRISPR-Cas9 technology has transformed the field of genetic engineering by providing unprecedented precision in genome editing and creating new opportunities for therapeutic interventions. This technology comprises a versatile genome-editing toolkit for achieving a variety of therapeutic objectives. In the context of leukemia, a group of life-threatening hematologic malignancies, CRISPR-Cas9 has emerged as a transformative tool for immunotherapy. By enabling targeted modifications of immune cells, such as T cells, this technology enhances their ability to recognize and eradicate leukemic cells. CRISPR-Cas9 facilitates the disruption of immune checkpoint inhibitors, the insertion of chimeric antigen receptors (CARs), and the correction of genetic mutations that drive leukemia progression. These advancements have led to the development of more potent and personalized immunotherapies, such as CAR-T cell therapies, with improved efficacy and reduced off-target effects. Moreover, CRISPR-Cas9 allows researchers to model leukemia in vitro, providing deeper insights into disease mechanisms and accelerating the discovery of novel therapeutic targets. Despite challenges such as delivery efficiency and potential immunogenicity, the integration of CRISPR-Cas9 into leukemia immunotherapy represents a paradigm shift, offering hope for durable remissions and potentially curative outcomes. As clinical trials progress, this technology promises to redefine the standard of care for leukemia patients, ushering in a new era of precision medicine. This narrative review explores the revolutionary applications of CRISPR-Cas9 in redefining therapeutic strategies for leukemia.
与碱基编辑器、引导编辑器和其他核酸酶平台一起，CRISPR-Cas9技术通过在基因组编辑中提供前所未有的精确性以及为治疗干预创造新机会，改变了基因工程领域。这项技术包括一个多功能的基因组编辑工具包，可实现多种治疗目标。在白血病（一类危及生命的血液系统恶性肿瘤）的背景下，CRISPR-Cas9已成为免疫治疗的变革性工具。通过对免疫细胞（如T细胞）进行靶向修饰，该技术增强了它们识别和根除白血病细胞的能力。CRISPR-Cas9有助于破坏免疫检查点抑制剂、插入嵌合抗原受体（CAR）以及纠正驱动白血病进展的基因突变。这些进展导致了更有效和个性化的免疫疗法（如CAR-T细胞疗法）的发展，其疗效得到改善，脱靶效应降低。此外，CRISPR-Cas9使研究人员能够在体外对白血病进行建模，从而更深入地了解疾病机制并加速新型治疗靶点的发现。尽管存在诸如递送效率和潜在免疫原性等挑战，但将CRISPR-Cas9整合到白血病免疫治疗中代表了一种范式转变，为持久缓解和潜在的治愈结果带来了希望。随着临床试验的推进，这项技术有望重新定义白血病患者的护理标准，迎来精准医学的新时代。这篇叙述性综述探讨了CRISPR-Cas9在重新定义白血病治疗策略方面的革命性应用。
