Current Status of Gene Therapy for Brain Tumors

Jianfang Ning, Samuel D. Rabkin

Research output: Chapter in Book/Report/Conference proceedingChapter

3 Scopus citations


Brain tumors, solid neoplasms within the brain or the central spinal canal, can often be debilitating and difficult to treat. Glioblastoma (GBM) is the most aggressive and deadliest primary brain tumor in adults, not responding to current therapies and with a very low percentage of 5-year survivors. Therefore, the development of new treatments is critical. Gene therapy is a treatment strategy that relies on the delivery of genetic material into cells for therapeutic purposes, and has demonstrated increasing success in preclinical models and clinical trials. Within this strategy, we include selectively replication-competent oncolytic viruses. While the oncolytic viruses act directly as antitumor agents due to virus replication, the effects are due to the transfer of viral genetic material to the cells. GBM is a good target for gene therapy because tumors remain locally within the brain and only rarely metastasize to other tissues; the majority of cells in the brain are postmitotic, which allows for specific targeting of dividing tumor cells; and tumors can often be accessed neurosurgically for administration of therapy. This chapter will focus on GBM and describe current gene therapy strategies used for brain tumors, including cytotoxic, immune-stimulatory, tumor microenvironment disrupting gene therapy, oncolytic viruses, small RNA interference, and delivery systems encompassing viruses, stem cells, and nanotechnology, as well as current challenges and future directions.

Original languageEnglish (US)
Title of host publicationTranslating Gene Therapy to the Clinic
Subtitle of host publicationTechniques and Approaches
PublisherElsevier Inc.
Number of pages19
ISBN (Electronic)9780128005644
ISBN (Print)9780128005637
StatePublished - 2015
Externally publishedYes

Bibliographical note

Publisher Copyright:
© 2015 Elsevier Inc. All rights reserved..

Copyright 2020 Elsevier B.V., All rights reserved.


  • Clinical trials
  • Glioblastoma
  • Oncolytic virus
  • Stem cells
  • Suicide genes
  • Virus vectors


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