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利用不断演进的 CRISPR/Cas9 用于精准肿瘤学

英文原题:Harnessing the evolving CRISPR/Cas9 for precision oncology.

查看英文原题

Harnessing the evolving CRISPR/Cas9 for precision oncology.

PubMed 2024/08/08(内容时间) J Transl Med Q1 · IF 9.7(JCR 2025)

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中文摘要

规律成簇间隔短回文重复序列(CRISPR)/Cas9系统是基因工程领域的突破性创新,彻底改变了应对复杂疾病的方式,并促成CASGEVY获批用于镰状细胞贫血。CRISPR/Cas9源自微生物免疫防御机制,具有精准、灵活和通用等基因编辑特点,已成为在哺乳动物中精准操作DNA的多用途工具。在实践应用过程中,新型直系同源物和变体不断被开发利用。这有助于理解疾病本质,尤其是癌症,对其诊断、预防和治疗至关重要。CRISPR/Cas9不仅用于研究肿瘤基因功能,也用于构建不同癌症模型,为理解肿瘤生物学、耐药和免疫逃逸提供重要见解。在癌症治疗中,CRISPR/Cas9有助于开发个体化精准疗法,可选择性激活或关闭肿瘤细胞内的基因,以抑制肿瘤生长和侵袭并提高癌细胞对治疗的敏感性。

此外,该技术促进创新疗法开发,例如提高重编程免疫细胞的靶向效率,CAR-T 方案的进展即为一例。除治疗外,它还是筛查易感基因的有力工具,可望在肿瘤发生或进展前进行干预。

然而,尽管CRISPR/Cas9潜力巨大,其在癌症研究和治疗中的应用仍面临疗效、效率、技术和安全方面的重要考量,需要持续技术创新加以解决。CRISPR/Cas9系统正在推动癌症研究和治疗变革,为深入理解和管理癌症开辟新途径。将这项持续发展的技术融入临床实践,有望开启精准肿瘤学新时代,为癌症患者带来靶向、个体化且可能治愈的疗法。

展开英文摘要原文

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.

论文信息

作者
Li T、Li S、Kang Y、Zhou J、Yi M
第一作者单位
Department of Gynecology, The Second Affiliated Hospital, Zhejiang University School of Medicine, Zhejiang University, Hangzhou, 310009, People's Republic of China.China
通讯作者单位
Department of Breast Surgery, The First Affiliated Hospital, College of Medicine, Zhejiang University, Hangzhou, 310000, People's Republic of China. 1322068@zju.edu.cn.China
文献类型
综述 · 非美国政府资助研究
期刊
Journal of translational medicine2024 Aug 8
原文标识
PubMed 39118151 · DOI 10.1186/s12967-024-05570-4