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

Abstract

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
Pages (from-to)685-701
Number of pages17
JournalExpert Review of Neurotherapeutics
Volume3
Issue number5
DOIs
StatePublished - Sep 2003
Externally publishedYes

Bibliographical note

Funding Information:
The authors found the following Internet websites very useful in providing useful and accurate information about neuro-oncological applications for gene therapy.  www.clinicaltrials.gov (Accessed June 2003). A website pro-vided by the National Institutes of Health and maintained by the National Library of Medicine listing all active clinical research studies.  www.ncbi.nlm.nih.gov/entrez/queryfcgi (Accessed June 2003). This Pubmed website, maintained by the National Library of Medicine, provides a database search for over 12 million MEDLINE citations from all the major medical journals dat-ing back to the 1960s.  www.nci.nih.gov (Cancer.gov) (Accessed June 2003). The

Funding Information:
 www.nabtt.org (Accessed June 2003). The official website for the New Approaches to Brain Tumor Treatment Consor-tium, a National Cancer Institute funded multi-institutional consortium dedicated to malignant brain tumor research.

Funding

The authors found the following Internet websites very useful in providing useful and accurate information about neuro-oncological applications for gene therapy.  www.clinicaltrials.gov (Accessed June 2003). A website pro-vided by the National Institutes of Health and maintained by the National Library of Medicine listing all active clinical research studies.  www.ncbi.nlm.nih.gov/entrez/queryfcgi (Accessed June 2003). This Pubmed website, maintained by the National Library of Medicine, provides a database search for over 12 million MEDLINE citations from all the major medical journals dat-ing back to the 1960s.  www.nci.nih.gov (Cancer.gov) (Accessed June 2003). The  www.nabtt.org (Accessed June 2003). The official website for the New Approaches to Brain Tumor Treatment Consor-tium, a National Cancer Institute funded multi-institutional consortium dedicated to malignant brain tumor research.

FundersFunder number
National Institutes of Health
National Cancer Institute
U.S. National Library of Medicine

    Keywords

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

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