Somatic Gene Therapy for Nervous System Disease

  1. Gregory R. Bock Organizer and
  2. Jamie A. Goode
  1. Mark H. Tuszynski1,2 and
  2. Fred H. Gage3

Published Online: 28 SEP 2007

DOI: 10.1002/9780470514863.ch7

Ciba Foundation Symposium 196 - Growth Factors as Drugs for Neurological and Sensory Disorders

Ciba Foundation Symposium 196 - Growth Factors as Drugs for Neurological and Sensory Disorders

How to Cite

Tuszynski, M. H. and Gage, F. H. (2007) Somatic Gene Therapy for Nervous System Disease, in Ciba Foundation Symposium 196 - Growth Factors as Drugs for Neurological and Sensory Disorders (eds G. R. Bock and J. A. Goode), John Wiley & Sons, Ltd., Chichester, UK. doi: 10.1002/9780470514863.ch7

Author Information

  1. 1

    Department of Neuroscience, University of California at San Diego, 9500 Gilman Drive, La Jolla, CA 92093

  2. 2

    Veterans Affairs Medical Center, San Diego, CA 92162, USA

  3. 3

    Salk Institute for Biological Studies, La Jolla, CA 92037, USA

Publication History

  1. Published Online: 28 SEP 2007

ISBN Information

Print ISBN: 9780471957218

Online ISBN: 9780470514863

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Keywords:

  • somatic gene therapy;
  • neurotrophic factors;
  • maturation;
  • gene therapy;
  • peripheral administration

Summary

Neurotrophic factors are target-derived molecules that prevent neuronal degeneration during development and, in some cases, during adulthood. They offer substantial promise as therapeutic agents in neurological disease by preventing cell loss and promoting axonal regeneration. However, the optimal means of delivering neurotrophic factors to the nervous system, and the CNS in particular, is an unresolved issue. Neurotrophic factors rarely influence only a single target neuronal population, hence broad delivery of neurotrophic factors to the nervous system may result in effects on multiple non-targeted neuronal populations. Ideally, neurotrophin delivery to the nervous system should be target-specific, regionally restricted, chronic, safe, well-tolerated and of sufficient concentration to elicit responses from target neurons. In this paper we discuss the use of somatic gene transfer methods to deliver neurotrophic factors to the CNS in a manner that seeks to meet the above criteria.