成簇规则间隔短回文重复 (CRISPR)/Cas9 系统是基因工程领域的一项突破性创新，彻底改变了我们克服复杂疾病的方法，最终导致 CASGEVY™ 被批准用于治疗镰状细胞性贫血。 CRISPR/Cas9源自微生物免疫防御机制，具有基因编辑的精确性、可操作性和通用性，已成为精确操作哺乳动物DNA的多功能工具。在将其应用于实践的过程中，对新的直系同源物和变体的不断开发从未停止。它有利于了解疾病特别是癌症的本质，对诊断、预防和治疗至关重要。 CRISPR/Cas9 不仅用于研究肿瘤基因的功能，还用于模拟不同的癌症，为肿瘤生物学、耐药性和免疫逃避提供有价值的见解。在癌症治疗中，CRISPR/Cas9有助于开发个体化和精确的癌症疗法，可以选择性地激活或失活肿瘤细胞内的基因，旨在削弱肿瘤的生长和侵袭并使癌细胞对治疗敏感。此外，它还促进创新疗法的开发，提高重编程免疫细胞的靶向效率，CAR-T疗法的进步就是例证。除了治疗之外，它还是筛查易感基因的有效工具，提供了在肿瘤发生或进展之前进行干预的可能性。然而，尽管其潜力巨大，但 CRISPR/Cas9 在癌症研究和治疗中的应用伴随着显着的功效、效率、技术和安全性考虑。需要不断升级的技术创新来解决这些问题。 CRISPR/Cas9 系统正在彻底改变癌症研究和治疗，为我们对癌症的理解和管理开辟新途径。这种不断发展的技术与临床实践的整合预示着精准肿瘤学的新时代，为癌症患者提供有针对性的、个性化的和潜在的治愈性治疗。© 2024。作者。

The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)/Cas9 system, a groundbreaking innovation in genetic engineering, has revolutionized our approach to surmounting complex diseases, culminating in CASGEVY™ approved for sickle cell anemia. Derived from a microbial immune defense mechanism, CRISPR/Cas9, characterized as precision, maneuverability and universality in gene editing, has been harnessed as a versatile tool for precisely manipulating DNA in mammals. In the process of applying it to practice, the consecutive exploitation of novel orthologs and variants never ceases. It's conducive to understanding the essentialities of diseases, particularly cancer, which is crucial for diagnosis, prevention, and treatment. CRISPR/Cas9 is used not only to investigate tumorous genes functioning but also to model disparate cancers, providing valuable insights into tumor biology, resistance, and immune evasion. Upon cancer therapy, CRISPR/Cas9 is instrumental in developing individual and precise cancer therapies that can selectively activate or deactivate genes within tumor cells, aiming to cripple tumor growth and invasion and sensitize cancer cells to treatments. Furthermore, it facilitates the development of innovative treatments, enhancing the targeting efficiency of reprogrammed immune cells, exemplified by advancements in CAR-T regimen. Beyond therapy, it is a potent tool for screening susceptible genes, offering the possibility of intervening before the tumor initiative or progresses. However, despite its vast potential, the application of CRISPR/Cas9 in cancer research and therapy is accompanied by significant efficacy, efficiency, technical, and safety considerations. Escalating technology innovations are warranted to address these issues. The CRISPR/Cas9 system is revolutionizing cancer research and treatment, opening up new avenues for advancements in our understanding and management of cancers. The integration of this evolving technology into clinical practice promises a new era of precision oncology, with targeted, personalized, and potentially curative therapies for cancer patients.© 2024. The Author(s).