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Updated: Aug 30, 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 for cancer and metastatic disease
Susan B Kesmodel1, Francis R Spitz
1Department of Surgery, Hospital of the University of Pennsylvania, 4 Silverstein, 3400 Spruce Street, Philadelphia, PA 19104, USA. kesmodes@uphs.upenn.edu
Abstract:
Gene therapy has been applied to the treatment of cancer and metastatic disease for over ten years. Research in this area has utilised multiple gene therapy approaches including targeting tumour suppressor genes and oncogenes, stimulating the immune system, targeted chemotherapy, antiangiogenic strategies, and direct viral oncolysis. In recent years, gene delivery vectors have been developed that selectively target tumour cells through tumour-specific receptors, deletion of certain viral gene sequences, or incorporation of tumour-specific promoter sequences that drive gene expression. Preclinical models have produced promising results, demonstrating significant tumour regression and reduction of metastatic disease. Unfortunately, only limited responses have been observed in clinical trials. The main limitations in treating metastatic disease include poor vector transduction efficiencies and difficulties in targeting remote tumour cells with systemic vector delivery. Currently, various groups are investigating means to improve gene delivery and clinical responses by continuing to modify gene delivery vectors and by concentrating on combination gene therapy and multimodality therapy.
Insights
Gene therapy shows promise for cancer treatment, but clinical trials face challenges. Researchers are improving gene delivery vectors and exploring combination therapies to enhance effectiveness against metastatic disease.
Area of Science:
- Oncology
- Molecular Biology
- Biotechnology
Background:
- Gene therapy has been utilized for over a decade in treating cancer and metastatic disease.
- Approaches include targeting oncogenes/tumor suppressor genes, immune stimulation, chemotherapy, antiangiogenesis, and viral oncolysis.
Purpose of the Study:
- To review advancements in gene therapy for cancer, focusing on vector development and clinical limitations.
- To highlight ongoing research aimed at improving gene delivery and therapeutic outcomes.
Main Methods:
- Development of gene delivery vectors targeting tumor cells via specific receptors or promoters.
- Utilizing preclinical models to assess tumor regression and metastatic disease reduction.
Main Results:
- Preclinical studies demonstrate significant tumor regression and reduced metastatic disease.
- Clinical trials have shown limited responses, indicating challenges in efficacy.
Conclusions:
- Key limitations include poor vector transduction and difficulty targeting remote tumor cells.
- Ongoing research focuses on modifying vectors and employing combination/multimodality therapies for improved clinical responses.
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