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Gene therapy as a potential tool for treating neuroblastoma-a focused review
M D Kumar1, A Dravid1, A Kumar1
1School of Biosciences and Technology, Vellore Institute of Technology University, Vellore, Tamil Nadu, India.
Abstract:
Neuroblastoma, a solid tumor caused by rapid division of undifferentiated neuroblasts, is the most common childhood malignancy affecting children aged <5 years. Several approaches and strategies developed and tested to cure neuroblastoma have met with limited success due to different reasons. Many oncogenes are deregulated during the onset and development of neuroblastoma and thus offer an opportunity to circumvent this disease if the expression of these genes is restored to normalcy. Gene therapy is a powerful tool with the potential to inhibit the deleterious effects of oncogenes by inserting corrected/normal genes into the genome. Both viral and non-viral vector-based gene therapies have been developed and adopted to deliver the target genes into neuroblastoma cells. These attempts have given hope to bringing in a new regime of treatment against neuroblastoma. A few gene-therapy-based treatment strategies have been tested in limited clinical trials yielding some positive results. This mini review is an attempt to provide an overview of the available options of gene therapy to treat neuroblastoma.
Insights
Gene therapy offers a promising approach to treat neuroblastoma, a common childhood cancer. By correcting deregulated oncogenes, gene therapy aims to restore normal gene expression and combat this malignancy.
Area of Science:
- Oncology
- Genetics
- Pediatric Medicine
Background:
- Neuroblastoma is a frequent childhood cancer in those under 5, originating from undifferentiated neuroblasts.
- Current treatments for neuroblastoma have shown limited success, necessitating novel therapeutic strategies.
- Aberrant oncogene expression is a key factor in neuroblastoma development and progression.
Purpose of the Study:
- To review the potential of gene therapy as a treatment modality for neuroblastoma.
- To explore how gene therapy can address oncogene deregulation in neuroblastoma.
- To provide an overview of current gene therapy options for neuroblastoma treatment.
Main Methods:
- Review of existing literature on gene therapy applications in neuroblastoma.
- Analysis of viral and non-viral vector systems for gene delivery in neuroblastoma cells.
- Examination of clinical trial data for gene therapy-based neuroblastoma treatments.
Main Results:
- Gene therapy demonstrates potential in inhibiting oncogene effects by introducing normal genes.
- Both viral and non-viral vectors are being utilized for gene delivery to neuroblastoma cells.
- Early clinical trials of gene therapy for neuroblastoma have shown encouraging outcomes.
Conclusions:
- Gene therapy presents a hopeful new treatment avenue for neuroblastoma.
- Restoring normal gene expression via gene therapy could overcome treatment limitations.
- Further research and clinical trials are warranted to fully establish gene therapy's role in neuroblastoma care.
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