Chapter 20. Applications of Gene Therapy to Neurological Diseases and Injuries

  1. Peter J. Quesenberry3,
  2. Gary S. Stein3,
  3. Bernard G. Forget4,
  4. Sherman M. Weissman4
  1. Derek L. Choi-Lundberg1,
  2. Martha C. Bohn2

Published Online: 13 MAY 2002

DOI: 10.1002/0471223956.ch20

Book Title

Stem Cell Biology and Gene Therapy

Stem Cell Biology and Gene Therapy

Additional Information

How to Cite

Choi-Lundberg, D. L. and Bohn, M. C. (2002) Applications of Gene Therapy to Neurological Diseases and Injuries, in Stem Cell Biology and Gene Therapy (eds P. J. Quesenberry, G. S. Stein, B. G. Forget and S. M. Weissman), John Wiley & Sons, Inc., New York, USA. doi: 10.1002/0471223956.ch20

Editor Information

  1. 3

    University of Massachusetts, Worcester, Massachusetts

  2. 4

    Yale University School of Medicine, New Haven, Connecticut

Author Information

  1. 1

    Department of Neurobiology and Anatomy, Box 603, 601 Elmwood Ave, University of Rochester School of Medicine and Dentistry, Rochester, NY 14642

  2. 2

    Children's Memorial Institute for Education and Research, Department of Pediatrics, Northwestern University Medical School, Chicago, IL 60614

Publication History

  1. Published Online: 13 MAY 2002
  2. Published Print: 27 AUG 1998

ISBN Information

Print ISBN: 9780471146568

Online ISBN: 9780471223955

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Keywords:

  • neurodegeneration;
  • gene therapy;
  • applications;
  • neurotrophic factor therapies;
  • delivery systems;
  • central nervous system;
  • Parkinson's disease;
  • motoneuron disorders;
  • Alzheimer's disease;
  • CAG repeat expansion disorders;
  • stroke;
  • apoptosis;
  • peripheral nervous system disorders;
  • spinal cord trauma

Summary

The concept of gene therapy has been expanded to include molecular genetic techniques to deliver therapeutic molecules for the treatment of any disease, genetic or nongenetic. Potential applications of gene therapy for the nervous system include correction of inherited errors of metabolism that affect the nervous system, delivery of toxic gene products to brain tumors, delivery of neuroprotective molecules to neuronal populations undergoing degeneration, and replacement of neurotransmitter-synthesizing enzymes. Such applications are the subject of this chapter.

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