Shah Smit, Green Joshua, Graff Shantelle A, Li Qi, Heiss John D
Neurology Department, School of Medicine, University of South Carolina, 15 Medical Park Rd., Columbia, SC 29203, USA.
Surgical Neurology Branch, NINDS, NIH 10 Center Drive, Bethesda, MD 20892, USA.
Viruses. 2025 Jan 16;17(1):118. doi: 10.3390/v17010118.
Glioblastoma multiforme (GBM) is a devastating, aggressive primary brain tumor with poor patient outcomes and a five-year survival of less than 10%. Significant limitations to effective GBM treatment include poor drug delivery across the blood-brain barrier, drug resistance, and complex genetic tumor alterations. Gene therapy uses a mechanism different from other GBM therapies to reduce tumor growth and enhance antitumor immunity. This review article will provide an update on various viral and nonviral vectors, their DNA and RNA cargoes, and how they genetically modify tumor cells and evoke therapeutic responses to GBM. The article explores the oncolytic and immunogenic effects of gene therapy agents. It reviews promising DNA transgenes, RNA inhibitors, and vectors for anti-GBM therapy. The possible benefits of combining gene therapy with standard GBM treatments will also be covered.
多形性胶质母细胞瘤（GBM）是一种极具破坏性的侵袭性原发性脑肿瘤，患者预后较差，五年生存率低于10%。有效治疗GBM的显著局限性包括药物难以透过血脑屏障、耐药性以及肿瘤复杂的基因改变。基因治疗采用与其他GBM治疗方法不同的机制来减少肿瘤生长并增强抗肿瘤免疫力。这篇综述文章将提供关于各种病毒和非病毒载体、它们的DNA和RNA载物，以及它们如何对肿瘤细胞进行基因改造并引发对GBM的治疗反应的最新信息。文章探讨了基因治疗药物的溶瘤和免疫原性作用。它综述了用于抗GBM治疗的有前景的DNA转基因、RNA抑制剂和载体。还将涵盖基因治疗与标准GBM治疗相结合的可能益处。
