Gene therapy for malignant brain tumors

Gustavo Pradilla, Tony Azzam, Paul P. Wang, Abraham J. Domb, Henry Brem

Research output: Contribution to journalReview articlepeer-review

1 Scopus citations


Malignant primary and metastatic brain tumors have remained fatal in spite of major advances in diagnostic tools and the improvement of conventional therapies. Recent discoveries in the molecular basis of the disease have allowed increased understanding of the events that lead to the development of brain tumors and have also brought a new spectrum of alternatives for treatment. By using gene therapy, brain tumors can be treated by targeting their fundamental molecular defects, delivering gene-drugs to the malignant cells. The possible targets for this type of treatment are progressively increasing but abundant clinical success has yet to be obtained, in part due to imperfect delivery systems. In this review, the genetic fundamentals of various cerebral neoplasms and neurogenetic syndromes, different strategies used for gene therapy, various available DNA delivery systems, status of ongoing clinical trials, and possible prospects for the future are discussed.

Original languageEnglish (US)
Pages (from-to)685-701
Number of pages17
JournalExpert review of neurotherapeutics
Issue number5
StatePublished - Sep 2003


  • Brain tumors
  • Gene delivery
  • Gene targeting
  • Gene therapy
  • Gliomas

ASJC Scopus subject areas

  • General Neuroscience
  • Clinical Neurology
  • Pharmacology (medical)


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