Gene Therapy for Glioblastoma Multiforme

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 tumo...

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Main Authors: Smit Shah, Joshua Green, Shantelle A. Graff, Qi Li, John D. Heiss
Format: Article
Language:English
Published: MDPI AG 2025-01-01
Series:Viruses
Subjects:
Online Access:https://www.mdpi.com/1999-4915/17/1/118
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author Smit Shah
Joshua Green
Shantelle A. Graff
Qi Li
John D. Heiss
author_facet Smit Shah
Joshua Green
Shantelle A. Graff
Qi Li
John D. Heiss
author_sort Smit Shah
collection DOAJ
description 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.
format Article
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series Viruses
spelling doaj-art-ab9aface0e984c9e9809c562ec4804d22025-01-24T13:52:39ZengMDPI AGViruses1999-49152025-01-0117111810.3390/v17010118Gene Therapy for Glioblastoma MultiformeSmit Shah0Joshua Green1Shantelle A. Graff2Qi Li3John D. Heiss4Neurology Department, School of Medicine, University of South Carolina, 15 Medical Park Rd., Columbia, SC 29203, USASurgical Neurology Branch, NINDS, NIH 10 Center Drive, Bethesda, MD 20892, USASurgical Neurology Branch, NINDS, NIH 10 Center Drive, Bethesda, MD 20892, USANeuro-Oncology Branch, CCR, NCI, NIH, 37 Convent Drive, Bethesda, MD 20892, USASurgical Neurology Branch, NINDS, NIH 10 Center Drive, Bethesda, MD 20892, USAGlioblastoma 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.https://www.mdpi.com/1999-4915/17/1/118glioblastomagenetherapyvectorsurvival
spellingShingle Smit Shah
Joshua Green
Shantelle A. Graff
Qi Li
John D. Heiss
Gene Therapy for Glioblastoma Multiforme
Viruses
glioblastoma
gene
therapy
vector
survival
title Gene Therapy for Glioblastoma Multiforme
title_full Gene Therapy for Glioblastoma Multiforme
title_fullStr Gene Therapy for Glioblastoma Multiforme
title_full_unstemmed Gene Therapy for Glioblastoma Multiforme
title_short Gene Therapy for Glioblastoma Multiforme
title_sort gene therapy for glioblastoma multiforme
topic glioblastoma
gene
therapy
vector
survival
url https://www.mdpi.com/1999-4915/17/1/118
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AT johndheiss genetherapyforglioblastomamultiforme