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Updated: Nov 19, 2025
Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
Published on: March 16, 2022
Adeno-associated virus (AAV)-based gene therapy for glioblastoma
Xin Xu1, Wenli Chen2, Wenjun Zhu3
1School of Medicine, Jiangsu University, Zhenjiang, 212013, Jiangsu, China. xinxin_8912@hotmail.com.
Abstract:
Glioblastoma (GBM) is the most common and malignant Grade IV primary craniocerebral tumor caused by glial cell carcinogenesis with an extremely poor median survival of 12-18 months. The current standard treatments for GBM, including surgical resection followed by chemotherapy and radiotherapy, fail to substantially prolong survival outcomes. Adeno-associated virus (AAV)-mediated gene therapy has recently attracted considerable interest because of its relatively low cytotoxicity, poor immunogenicity, broad tissue tropism, and long-term stable transgene expression. Furthermore, a range of gene therapy trials using AAV as vehicles are being investigated to thwart deadly GBM in mice models. At present, AAV is delivered to the brain by local injection, intracerebroventricular (ICV) injection, or systematic injection to treat experimental GBM mice model. In this review, we summarized the experimental trials of AAV-based gene therapy as GBM treatment and compared the advantages and disadvantages of different AAV injection approaches. We systematically introduced the prospect of the systematic injection of AAV as an approach for AAV-based gene therapy for GBM.
Insights
Adeno-associated virus (AAV) gene therapy shows promise for treating glioblastoma (GBM), a deadly brain cancer. This review compares AAV delivery methods, highlighting systemic injection as a potential approach for GBM treatment.
Area of Science:
- Neuro-oncology
- Gene Therapy
- Viral Vectors
Background:
- Glioblastoma (GBM) is a highly aggressive Grade IV primary brain tumor with a median survival of 12-18 months.
- Current treatments like surgery, chemotherapy, and radiotherapy offer limited survival benefits for GBM patients.
Purpose of the Study:
- To review experimental trials of adeno-associated virus (AAV)-based gene therapy for glioblastoma (GBM).
- To compare the advantages and disadvantages of different AAV delivery methods in preclinical GBM models.
- To explore the potential of systemic AAV injection for GBM gene therapy.
Main Methods:
- Systematic review of published experimental studies on AAV-mediated gene therapy for GBM.
- Analysis of different AAV administration routes: local injection, intracerebroventricular (ICV) injection, and systemic injection.
- Comparison of efficacy, safety, and feasibility of various AAV delivery strategies in GBM mouse models.
Main Results:
- AAV-mediated gene therapy is being investigated for GBM due to its low cytotoxicity, immunogenicity, and stable expression.
- Various AAV delivery methods are employed in experimental GBM models, each with specific benefits and drawbacks.
- Systemic AAV injection is presented as a promising approach for widespread brain delivery in GBM gene therapy.
Conclusions:
- AAV-based gene therapy represents a potential therapeutic strategy for glioblastoma.
- Understanding the nuances of different AAV delivery methods is crucial for optimizing treatment efficacy.
- Systemic AAV injection warrants further investigation as a viable route for GBM gene therapy.
