Chapter 9. Delivery Systems for Gene Therapy: Adeno-Associated Virus

  1. Peter J. Quesenberry3,
  2. Gary S. Stein3,
  3. Bernard G. Forget4,
  4. Sherman M. Weissman4
  1. Gabriele Kroner-Lux1,
  2. Christopher E. Walsh2,
  3. Richard Jude Samulski2

Published Online: 13 MAY 2002

DOI: 10.1002/0471223956.ch9

Book Title

Stem Cell Biology and Gene Therapy

Stem Cell Biology and Gene Therapy

Additional Information

How to Cite

Kroner-Lux, G., Walsh, C. E. and Jude Samulski, R. (2002) Delivery Systems for Gene Therapy: Adeno-Associated Virus, 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.ch9

Editor Information

  1. 3

    University of Massachusetts, Worcester, Massachusetts

  2. 4

    Yale University School of Medicine, New Haven, Connecticut

Author Information

  1. 1

    University of North Carolina at Chapel Hill, Gene Therapy Center, CB#7352, 7109 Thurston-Bowles, Chapel Hill, NC 27599

  2. 2

    University of North Carolina at Chapel Hill, Gene Therapy Center, 7109 Thurston-Bowles, Chapel Hill, NC 27599

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:

  • gene therapy;
  • delivery systems;
  • adeno-associated virus (AAV) vectors;
  • AAV-mediated transduction;
  • marker genes;
  • hematopoietic cells;
  • integration;
  • therapeutic genes

Summary

The purpose of this chapter is to provide an overview of recombinant adeno-associated virus (rAAV) vectors and the current “state of the art” of their potential utility in gene therapy of hematopoietic disorders. The authors briefly review AAV biology and genetics and suggest more general reviews about AAV. The basic requirements that need to be fulfilled for gene therapy as treatment of hematopoietic disorders are presented.

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