Chapter 13. Development of Gene Therapy for Gaucher Disease

  1. Peter J. Quesenberry2,
  2. Gary S. Stein2,
  3. Bernard G. Forget3,
  4. Sherman M. Weissman3
  1. J.A. Barranger,
  2. E.O. Rice,
  3. J. Dunigan,
  4. M. Eljanne,
  5. N. Takiyama,
  6. M. Nimgaonkar,
  7. J. Mierski,
  8. M. Beeler,
  9. A. Kemp,
  10. J. Lancia,
  11. S. Lucot,
  12. S. Schierer-Fochler,
  13. J. Mannion-Henderson,
  14. T. Mohney,
  15. W. Swaney,
  16. A. Bahnson,
  17. V. Bansal,
  18. E. Ball

Published Online: 13 MAY 2002

DOI: 10.1002/0471223956.ch13

Book Title

Stem Cell Biology and Gene Therapy

Stem Cell Biology and Gene Therapy

Additional Information

How to Cite

Barranger, J.A., Rice, E.O., Dunigan, J., Eljanne, M., Takiyama, N., Nimgaonkar, M., Mierski, J., Beeler, M., Kemp, A., Lancia, J., Lucot, S., Schierer-Fochler, S., Mannion-Henderson, J., Mohney, T., Swaney, W., Bahnson, A., Bansal, V. and Ball, E. (2002) Development of Gene Therapy for Gaucher Disease, 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.ch13

Editor Information

  1. 2

    University of Massachusetts, Worcester, Massachusetts

  2. 3

    Yale University School of Medicine, New Haven, Connecticut

Author Information

  1. University of Pittsburgh Medical Center, Pittsburgh, PA 15261

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:

  • Gaucher disease;
  • gene transfer therapy;
  • MFG-GC;
  • CD34+ cells;
  • transduction;
  • preclinical studies;
  • clinical trial;
  • hematopoietic cells;
  • prenatal gene therapy

Summary

The pathobiology of lysosomal storage disorders should dictate the target cell for gene transfer in the development of gene therapy. However, because the pathogenesis of these and most diseases is only partially defined, a variety of gene transfer approaches may need to be studied before a successful therapy is discovered (Table 1). Gaucher disease (GD) is the most common lysosomal storage disorder and is the focus of this chapter.

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