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Ex Vivo Gene Therapy in the Central Nervous System

  • Chapter
CNS Neuroprotection

Part of the book series: Handbook of Experimental Pharmacology ((HEP,volume 155))

Abstract

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.

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  1. A. Blesch
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  2. M. H. Tuszynski
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  1. Pfizer Global Research and Development, Ann Arbor Laboratories, 2800 Plymouth Road, Ann Arbor, MI, 48105, USA

    Frank W. Marcoux (Vice President, Biology) (Vice President, Biology)

  2. Department of Neurology Box 8111, Washington University School of Medicine, 660 South Euclid Avenue, St. Louis, MO, 63110, USA

    Dennis W. Choi

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Blesch, A., Tuszynski, M.H. (2002). Ex Vivo Gene Therapy in the Central Nervous System. In: Marcoux, F.W., Choi, D.W. (eds) CNS Neuroprotection. Handbook of Experimental Pharmacology, vol 155. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-662-06274-6_11

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