Department of Neurology, Division of Neuro-Oncology, Northwestern University, 710 N. Lake Shore Dr., Abbott Hall Room 1123, Chicago, IL, 60611, USA.
Curr Oncol Rep. 2017 Sep 2;19(11):69. doi: 10.1007/s11912-017-0628-z.
Glioblastoma multiforme (GBM) is the most common primary malignant brain tumor in adults with a dismal prognosis despite aggressive multimodal management thus novel treatments are urgently needed. Gene therapy is a versatile treatment strategy being investigated in multiple cancers including GBM. In gene therapy, a variety of vectors or "carriers" are used to deliver genes designed for different anti-tumoral effects. Gene delivery vehicles and approaches to treatment will be addressed in this review.
The most commonly studied vectors are viral based, however, driven by advances in biomedical engineering, mesenchymal and neural stem cells, as well as multiple different types of nanoparticles have been developed to improve tumor tropism and also increase gene transfer into tumor cells. Different genes have been studied including suicide genes, which convert non-toxic prodrug into cytotoxic drug; immunomodulatory genes, which stimulate the immune system; and tumor suppressor genes which repair the defect that allow cells to divide unchecked. Gene therapy may be a promising treatment strategy in neuro-oncology as it is versatile and flexible due to the ability to tailor vectors and genes for specific therapeutic activity. Pre-clinical studies and clinical trials have demonstrated feasibility and safety of gene therapy; however, further studies are required to determine efficacy.
多形性胶质母细胞瘤（GBM）是成年人中最常见的原发性恶性脑肿瘤，尽管采用了积极的多模式治疗，但预后仍然很差，因此迫切需要新的治疗方法。基因治疗是一种多用途的治疗策略，正在多种癌症包括 GBM 中进行研究。在基因治疗中，使用多种载体或“载体”来递送至肿瘤的基因，这些基因设计用于不同的抗肿瘤作用。本综述将讨论基因传递载体和治疗方法。
最常研究的载体是基于病毒的，然而，随着生物医学工程的进步，间充质和神经干细胞，以及多种不同类型的纳米粒子的发展，已经改善了肿瘤趋向性，并增加了基因转移到肿瘤细胞。已经研究了不同的基因，包括自杀基因，将非毒性前药转化为细胞毒性药物；免疫调节基因，刺激免疫系统；以及肿瘤抑制基因，修复允许细胞不受控制分裂的缺陷。基因治疗可能是神经肿瘤学中一种很有前途的治疗策略，因为它具有多功能性和灵活性，能够为特定的治疗活性定制载体和基因。临床前研究和临床试验已经证明了基因治疗的可行性和安全性；然而，需要进一步的研究来确定疗效。
