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

Cancer Gene Therapy (2025)Cite this article

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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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Fig. 1: The CRISPR/Cas tools.
Fig. 2: Applications of CRISPR technology in cancer therapy.
Fig. 3: Schematic diagram of strategies to overcome the limitations of conventional cancer treatments.
Fig. 4: CRISPR-based genome-wide screening.
Fig. 5: The CRISPR-engineered CAR-T therapy.

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Funding

This work was supported by a grant from Kyung Hee University in 2023 [KHU-20230920].

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  1. Department of Regulatory Science, Graduate School, Kyung Hee University, 26 Kyungheedae-ro, Dongdaemun-gu, Seoul, 02447, Republic of Korea

    Hyeonjeong Park & Taeyoung Koo

  2. College of Pharmacy and Institute of Integrated Pharmaceutical Sciences, Kyung Hee University, 26 Kyungheedae-ro, Dongdaemun-gu, Seoul, 02447, Republic of Korea

    Suyeun Yu & Taeyoung Koo

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TK designed, supervised, and wrote the manuscript. HP and SY provided conceptualization and wrote the manuscript. HP edited the manuscript and generated the figures and table.

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Correspondence to Taeyoung Koo.

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Park, H., Yu, S. & Koo, T. Gene editing in cancer therapy: overcoming drug resistance and enhancing precision medicine. Cancer Gene Ther (2025). https://doi.org/10.1038/s41417-025-00959-9

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