Chapter 16. Clinical Applications of Gene Therapy: Anemias

  1. Peter J. Quesenberry2,
  2. Gary S. Stein2,
  3. Bernard G. Forget3,
  4. Sherman M. Weissman3
  1. George F. Atweh1,
  2. Bernard G. Forget3

Published Online: 13 MAY 2002

DOI: 10.1002/0471223956.ch16

Book Title

Stem Cell Biology and Gene Therapy

Stem Cell Biology and Gene Therapy

Additional Information

How to Cite

Atweh, G. F. and Forget, B. G. (2002) Clinical Applications of Gene Therapy: Anemias, 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.ch16

Editor Information

  1. 2

    University of Massachusetts, Worcester, Massachusetts

  2. 3

    Yale University School of Medicine, New Haven, Connecticut

Author Information

  1. 1

    Professor of Medicine, Division of Hematology, Mount Sinai School of Medicine, One Gustav Levy Place, New York, NY 10029

  2. 3

    Yale University School of Medicine, New Haven, Connecticut

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:

  • anemias;
  • globin disorders;
  • gene therapy;
  • clinical applications

Summary

The β-globin gene was one of the first human genes to be used in pioneering gene transfer experiments. However, it soon became apparent that the application of this evolving gene transfer technology to globin genes posed some unique and formidable challenges. In this chapter, the authors discuss those challenges, the approaches that are being developed to overcome them, and the remaining hurdles to the successful application of corrective genetic therapy in the treatment of patients afflicted by these disorders.

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