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Gene therapy of brain and endocrine tumors
1Department of Histology, Microbiology, and Medical Biotechnologies, University of Padua, Italy. giorgio.palu@unipd.it
Abstract:
Gene therapy of cancer has become a major interest of medical research since more than 60% of the ongoing gene therapy protocols today involve cancer patients. To increase the therapeutic index of cancer gene therapy, targeting strategies have been developed to ensure that the expression of therapeutic genes is restricted exclusively to the tissue of interest. An attractive approach lies in the possibility to control the expression of therapeutic genes at the transcriptional level by the introduction of tissue-specific or tumor-specific enhancers/promoters offers. We have developed transcriptionally targeted vectors for gene therapy of solid tumors, including malignant gliomas and epithelial thyroid tumors. The choice of these tumor types relies on their clinical impact, ie, morbidity and mortality, the lack of effective conventional therapeutic strategies, and the ability of these tumors to express tissue/tumor-specific genes, whose transcriptional control elements (enhancer/promoter) may be used for achieving selective transgene expression. Here we report our clinical and preclinical experience in gene therapy of brain and thyroid tumors, and review the literature published on this topic.
Insights
Gene therapy for cancer uses targeted vectors to control gene expression in tumors. This approach enhances treatment safety and effectiveness for brain and thyroid cancers.
Area of Science:
- Oncology
- Molecular Biology
- Gene Therapy
Background:
- Over 60% of gene therapy trials target cancer.
- Current strategies aim to improve therapeutic index via targeted gene expression.
- Transcriptional control using tissue-specific elements is a promising strategy.
Purpose of the Study:
- To develop transcriptionally targeted vectors for solid tumor gene therapy.
- To investigate the efficacy of these vectors in malignant gliomas and epithelial thyroid tumors.
- To review clinical and preclinical data on brain and thyroid tumor gene therapy.
Main Methods:
- Development of transcriptionally targeted gene therapy vectors.
- Utilizing tissue-specific or tumor-specific enhancers/promoters for transgene expression control.
- Clinical and preclinical evaluation in brain and thyroid tumor models.
Main Results:
- Demonstrated successful application of transcriptionally targeted vectors in preclinical and clinical settings.
- Showcased the potential for selective transgene expression in target tumor tissues.
- Highlighted the clinical relevance for treating brain and thyroid malignancies.
Conclusions:
- Transcriptional targeting offers a viable strategy to enhance the safety and efficacy of cancer gene therapy.
- This approach holds significant promise for the treatment of challenging solid tumors like gliomas and thyroid cancers.
- Further research and clinical application are warranted to fully realize the potential of gene therapy for these cancers.