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Updated: Aug 15, 2026
Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy
Published on: October 4, 2019
Viral vectors as therapeutic agents for glioblastoma
1Massachusetts General Hospital and Harvard Medical School, Department of Neurosurgery, Molecular Neurosurgery Laboratory, Boston, MA 02114, USA. MAGHI@PARTNERS.ORG
Abstract:
Established treatments such as surgery, radiation and chemotherapy have not altered the median survival of glioblastoma, the most common malignant brain tumor. Since these failures reflect the highly invasive nature of glioblastoma, as well as the fact that few cells are actively replicating at any given point in time, therapies need to act in areas of the brain distant from the site of tumor origin and for long after their introduction. Over the past decade, laboratory studies and early clinical trials have raised hope that these therapeutic requirements may be fulfilled by gene therapy using non-replicating transgene-bearing viruses, oncolytic viruses or migratory stem cells to deliver tumoricidal transgenes. The principles behind these approaches and their initial results are reviewed.
Insights
Glioblastoma treatments like surgery, radiation, and chemotherapy are ineffective. Gene therapy using viruses or stem cells shows promise for treating this aggressive brain tumor.
Area of Science:
- Neuro-oncology
- Gene Therapy
- Oncolytic Viruses
Background:
- Glioblastoma is the most common malignant brain tumor.
- Established treatments (surgery, radiation, chemotherapy) have failed to improve median survival rates.
- Treatment challenges include glioblastoma's invasive nature and low cell replication rate.
Purpose of the Study:
- To review the principles and initial results of novel gene therapy approaches for glioblastoma.
- To explore the potential of viral and stem cell-based therapies for brain tumors.
Main Methods:
- Review of laboratory studies and early clinical trials.
- Focus on gene therapy vectors: non-replicating viruses, oncolytic viruses, and migratory stem cells.
- Delivery of tumoricidal transgenes to target glioblastoma cells.
Main Results:
- Gene therapy approaches show promise in preclinical and early clinical settings.
- Viral vectors and stem cells can deliver therapeutic genes to tumor sites.
- These novel therapies aim to overcome limitations of conventional treatments.
Conclusions:
- Gene therapy offers a potential new strategy for glioblastoma treatment.
- Further research and clinical trials are needed to optimize these approaches.
- Viral and stem cell-based gene therapies may fulfill the need for treatments that act distantly and persistently.
