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Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
Tumor-suppressing gene therapy
1Department of Thoracic and Cardiovascular Surgery, The University of Texas M.D. Anderson Cancer Center; Houston, Texas 77030, USA.
Abstract:
Tumor-suppressor genes play pivotal roles in maintaining genome integrity and in regulating cell proliferation, differentiation, and apoptosis. Their loss-of-function mutations are related directly to tumorigenesis. Thus, use of tumor-suppressor genes as anticancer therapeutics has been investigated rigorously in both experimental and clinical researches. Transfer of various tumor-suppressor genes directly to cancer cells has been demonstrated to suppress tumor growth via induction of apoptosis and cell-cycle arrest and, in some cases, with evidence for bystander effects. Various studies also have shown that combination of tumor-suppressor gene therapy with conventional anticancer therapy can yield synergistic therapeutic benefits. Clinical trials with tumor-suppressor genes, especially the p53 gene, have demonstrated that the treatment is well tolerated, and; favorable clinical responses, including a pathologically complete responses, have been observed in a subset of patients with advanced disease or with cancers resistant to conventional therapy. Yet, current gene replacement approaches in cancer gene therapy must be improved if they are to have a broader clinical impact. Efficient systemic gene delivery systems will be required ultimately for treatment of metastatic disease. In this review, we have recently summarized achievements in tumor-suppressor gene therapy with a focus on the p53 gene.
Insights
Tumor-suppressor gene therapy, particularly using the p53 gene, shows promise in cancer treatment by inducing apoptosis and cell-cycle arrest. Further advancements in gene delivery are needed for broader clinical impact, especially for metastatic diseases.
Area of Science:
- Oncology
- Molecular Biology
- Gene Therapy
Background:
- Tumor-suppressor genes are crucial for maintaining genome stability and regulating cell growth.
- Loss-of-function mutations in these genes are directly linked to cancer development.
- Gene therapy utilizing tumor-suppressor genes is a rigorously investigated anticancer therapeutic strategy.
Purpose of the Study:
- To review recent achievements in tumor-suppressor gene therapy.
- To highlight the role and potential of the p53 gene in cancer treatment.
- To discuss the challenges and future directions for improving gene replacement approaches.
Main Methods:
- Review of experimental and clinical research on tumor-suppressor gene therapy.
- Focus on studies involving direct transfer of tumor-suppressor genes to cancer cells.
- Analysis of clinical trial data, particularly for p53 gene therapy.
Main Results:
- Direct gene transfer suppresses tumor growth by inducing apoptosis and cell-cycle arrest.
- Combination therapy with conventional treatments can yield synergistic effects.
- Clinical trials with p53 gene therapy show good tolerability and favorable responses in some advanced or resistant cancers.
Conclusions:
- Tumor-suppressor gene therapy, especially with p53, is a promising approach with demonstrated clinical benefits.
- Current gene replacement strategies require improvement for wider clinical application.
- Development of efficient systemic gene delivery systems is essential for treating metastatic cancers.
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