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Updated: Jun 30, 2026

Testing Targeted Therapies in Cancer using Structural DNA Alteration Analysis and Patient-Derived Xenografts
Published on: July 25, 2020
Gene therapy for cancer
A A Gutierrez1, N R Lemoine, K Sikora
1Department of Clinical Oncology, Royal Postgraduate Medical School, Hammersmith Hospital, London, UK.
Abstract:
The molecular basis of cancer is now understood to involve activation of dominant oncogenes and inactivation of tumour suppressor genes, and these genetic events may represent novel targets for cancer therapy. This review focuses on the potential use and ethical implications of gene transfer to alter the behaviour of somatic cells in cancer patients. Antisense nucleic acids and ribozymes represent informational drugs that may be used to modulate the expression of selected genes and suppress malignant behaviour in cancer cells. Genetic immunomodulation by introducing genes for cytokines into cancer cells or lymphocytes can stimulate a cytotoxic immune response against the tumour. Gene transfer techniques can be applied to target prodrug activation specifically to tumour cells and also to protect normal tissues against toxic chemotherapy. Gene replacement therapy could even be used to restore the function of defective tumour suppressor genes.
Insights
Gene transfer offers novel cancer therapies by targeting oncogenes and tumor suppressor genes. Techniques like antisense nucleic acids, ribozymes, and gene replacement hold promise for suppressing cancer and enhancing immune response.
Area of Science:
- Oncology
- Molecular Biology
- Gene Therapy
Background:
- Cancer involves oncogene activation and tumor suppressor gene inactivation.
- Genetic events in cancer present novel therapeutic targets.
- Gene transfer offers potential strategies to modify somatic cells for cancer treatment.
Purpose of the Study:
- To review the potential applications and ethical considerations of gene transfer in cancer therapy.
- To explore how gene transfer can modulate gene expression and cellular behavior in cancer patients.
Main Methods:
- Review of current gene transfer technologies and their applications in oncology.
- Discussion of informational drugs (antisense nucleic acids, ribozymes) for gene expression modulation.
- Exploration of genetic immunomodulation and targeted prodrug activation strategies.
Main Results:
- Gene transfer can suppress malignant behavior by modulating oncogene and tumor suppressor gene expression.
- Genetic immunomodulation can enhance anti-tumor immune responses.
- Targeted gene transfer can improve chemotherapy efficacy and protect normal tissues.
Conclusions:
- Gene transfer represents a promising frontier in cancer therapy, targeting key genetic alterations.
- Ethical implications of somatic cell gene transfer require careful consideration.
- Future directions include gene replacement therapy for restoring tumor suppressor function.
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