Ex Vivo Gene Therapy in the Central Nervous System

  • A. Blesch
  • M. H. Tuszynski
Part of the Handbook of Experimental Pharmacology book series (HEP, volume 155)


The targeted delivery of genes into the adult central nervous system (CNS) has received considerable interest in recent years with the development of improved viral vector systems and suitable strategies for therapeutic intervention. Experimental gene therapy in animal models has been studied to prevent or slow the progression of chronic neurodegenerative diseases, to improve recovery after traumatic CNS injury and to kill malignant brain tumors. Genes that have been investigated in these various models include those that code for neurotrophic factors, neurotransmitter synthesis enzymes, modulators of neuronal and glial function, and inducers of cell death. Generally, two different types of gene therapy have been distinguished: in vivo and ex vivo gene therapy. The direct injection of genes into the CNS using viral vectors or DNA-liposome suspensions is termed in vivo gene therapy. Ex vivo gene therapy is based on genetic modification of cells in vitro followed by the grafting of these cells into the CNS. Ex vivo approaches to gene therapy will be the focus of this review.


Spinal Cord Injury Amyotrophic Lateral Sclerosis Gene Therapy Nerve Growth Factor Neurotrophic Factor 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


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© Springer-Verlag Berlin Heidelberg 2002

Authors and Affiliations

  • A. Blesch
  • M. H. Tuszynski

There are no affiliations available

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