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Updated: Mar 1, 2026

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Gene Therapy Used in Cancer Treatment
Thomas Wirth1, Seppo Ylä-Herttuala2,3
1Virtanen Institute, Department of Biotechnology and Molecular Medicine, University of Eastern Finland, Neulaniementie 2, 70211 Kuopio, Finland. thomas.wirth@uef.fi.
Abstract:
Cancer has been, from the beginning, a target of intense research for gene therapy approaches. Currently, more than 60% of all on-going clinical gene therapy trials worldwide are targeting cancer. Indeed, there is a clear unmet medical need for novel therapies. This is further urged by the fact that current conventional cancer therapies are frequently troubled by their toxicities. Different gene therapy strategies have been employed for cancer, such as pro-drug activating suicide gene therapy, anti-angiogenic gene therapy, oncolytic virotherapy, gene therapy-based immune modulation, correction/compensation of gene defects, genetic manipulation of apoptotic and tumor invasion pathways, antisense, and RNAi strategies. Cancer types, which have been targeted with gene therapy, include brain, lung, breast, pancreatic, liver, colorectal, prostate, bladder, head and neck, skin, ovarian, and renal cancer. Currently, two cancer gene therapy products have received market approval, both of which are in China. In addition, the stimulation of the host's immune system, using gene therapeutic approaches, has gained vast interest. The intention of this review is to point out the most commonly viral and non-viral vectors and methods used in cancer gene therapy, as well as highlight some key results achieved in clinical trials.
Insights
Gene therapy is a promising cancer treatment, with over 60% of trials targeting cancer. This review explores viral and non-viral vectors, methods, and clinical results for novel cancer therapies.
Area of Science:
- Oncology
- Gene Therapy
- Molecular Biology
Background:
- Cancer remains a significant global health challenge, necessitating novel therapeutic strategies beyond conventional treatments due to their frequent toxicities.
- Gene therapy has emerged as a key research area, with over 60% of ongoing clinical trials worldwide focused on cancer treatment.
- A clear unmet medical need exists for innovative cancer therapies with improved efficacy and reduced side effects.
Purpose of the Study:
- To provide a comprehensive overview of viral and non-viral vectors used in cancer gene therapy.
- To highlight various gene therapy strategies employed for different cancer types.
- To review key clinical trial results and advancements in cancer gene therapy.
Main Methods:
- Review of existing literature on gene therapy vectors and methods for cancer treatment.
- Analysis of gene therapy strategies including suicide gene therapy, anti-angiogenic therapy, oncolytic virotherapy, and immune modulation.
- Examination of clinical trial data for various cancer types, including brain, lung, breast, and pancreatic cancer.
Main Results:
- Gene therapy approaches encompass a wide range of strategies targeting diverse cancers.
- Viral and non-viral vectors are crucial for delivering therapeutic genes.
- Two gene therapy products for cancer have received market approval, both in China, indicating progress in the field.
Conclusions:
- Gene therapy offers a promising avenue for novel cancer treatments, addressing limitations of conventional therapies.
- The development and application of diverse gene therapy vectors and methods are advancing cancer care.
- Continued research and clinical trials are essential for realizing the full potential of gene therapy in oncology.
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