Gene editing in cancer therapy: overcoming drug resistance and enhancing precision medicine.

IF 5 3区医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Cancer gene therapy Pub Date : 2025-09-18 DOI:10.1038/s41417-025-00959-9

Hyeonjeong Park, Suyeun Yu, Taeyoung Koo

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引用次数: 0

Abstract

The CRISPR system has revolutionized cancer gene therapy, offering unparalleled precision in genetic manipulation for targeted oncogene disruption, mutation correction, and immune system modulation. This breakthrough tool has demonstrated remarkable potential in overcoming drug resistance, enhancing chemotherapy sensitivity, and improving immunotherapy strategies such as CRISPR-engineered CAR-T cells. Additionally, oncolytic virus-mediated CRISPR delivery has emerged as a novel approach for tumor-specific gene editing, minimizing off-target effects. The rapid transition of CRISPR-based cancer therapeutics from preclinical research to clinical trials underscores its therapeutic potential. This review explores the latest advancements in CRISPR applications for cancer therapy, including gene knockout, base editing for mutation correction, and integration with immune and viral therapies. Despite significant progress, challenges such as off-target effects, immune responses, and delivery limitations remain key hurdles. We discuss current strategies to enhance CRISPR safety and efficacy, emphasizing its potential for personalized cancer treatment.

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基因编辑在癌症治疗中的应用：克服耐药性，加强精准医疗。

CRISPR系统彻底改变了癌症基因治疗，为靶向癌基因破坏、突变纠正和免疫系统调节的基因操作提供了无与伦比的精确度。这一突破性工具在克服耐药性、增强化疗敏感性和改进免疫治疗策略（如crispr工程CAR-T细胞）方面显示出了巨大的潜力。此外，溶瘤病毒介导的CRISPR递送已经成为肿瘤特异性基因编辑的一种新方法，可以最大限度地减少脱靶效应。基于crispr的癌症治疗方法从临床前研究到临床试验的快速转变凸显了其治疗潜力。本文综述了CRISPR在癌症治疗中的最新应用进展，包括基因敲除、碱基编辑用于突变纠正，以及与免疫和病毒治疗的整合。尽管取得了重大进展，但脱靶效应、免疫反应和递送限制等挑战仍然是主要障碍。我们讨论了目前提高CRISPR安全性和有效性的策略，强调了其在个性化癌症治疗方面的潜力。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Cancer gene therapy 医学-生物工程与应用微生物

CiteScore

10.20

自引率

0.00%

发文量

150

审稿时长

4-8 weeks

期刊介绍： Cancer Gene Therapy is the essential gene and cellular therapy resource for cancer researchers and clinicians, keeping readers up to date with the latest developments in gene and cellular therapies for cancer. The journal publishes original laboratory and clinical research papers, case reports and review articles. Publication topics include RNAi approaches, drug resistance, hematopoietic progenitor cell gene transfer, cancer stem cells, cellular therapies, homologous recombination, ribozyme technology, antisense technology, tumor immunotherapy and tumor suppressors, translational research, cancer therapy, gene delivery systems (viral and non-viral), anti-gene therapy (antisense, siRNA & ribozymes), apoptosis; mechanisms and therapies, vaccine development, immunology and immunotherapy, DNA synthesis and repair. Cancer Gene Therapy publishes the results of laboratory investigations, preclinical studies, and clinical trials in the field of gene transfer/gene therapy and cellular therapies as applied to cancer research. Types of articles published include original research articles; case reports; brief communications; review articles in the main fields of drug resistance/sensitivity, gene therapy, cellular therapy, tumor suppressor and anti-oncogene therapy, cytokine/tumor immunotherapy, etc.; industry perspectives; and letters to the editor.