Adeno‐associated virus (AAV)-based gene therapy for glioblastoma

• Main Authors: Xin Xu, Wenli Chen, Wenjun Zhu, Jing Chen, Bin Ma, Jianxia Ding, Zaichuan Wang, Yifei Li, Yeming Wang, Xiaochun Zhang
• Format: Article
• Language: English
• Published: BMC 2021-01-01
• Series: Cancer Cell International
• Subjects: AAV; Glioblastoma; Gene therapy; Systemic injection; Local injection; Intracerebroventricular injection
• Online Access: https://doi.org/10.1186/s12935-021-01776-4

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.