Chapter 14. Clinical Applications of Gene Therapy: Correction of Genetic Disease Affecting Hematopoietic Cells

  1. Peter J. Quesenberry4,
  2. Gary S. Stein4,
  3. Bernard G. Forget5,
  4. Sherman M. Weissman5
  1. Jeffrey A. Medin1,
  2. Johan Richter2,
  3. Stefan Karlsson3

Published Online: 13 MAY 2002

DOI: 10.1002/0471223956.ch14

Book Title

Stem Cell Biology and Gene Therapy

Stem Cell Biology and Gene Therapy

Additional Information

How to Cite

Medin, J. A., Richter, J. and Karlsson, S. (2002) Clinical Applications of Gene Therapy: Correction of Genetic Disease Affecting Hematopoietic Cells, 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.ch14

Editor Information

  1. 4

    University of Massachusetts, Worcester, Massachusetts

  2. 5

    Yale University School of Medicine, New Haven, Connecticut

Author Information

  1. 1

    Section of Hematology/Oncology, University of Illinois at Chicago, Chicago, IL 60607-7173

  2. 2

    Gene Therapy Center, Molecular Medicine Section and The Department of Medicine, University Hospital, Lund, Sweden

  3. 3

    Gene Therapy Center, Molecular Medicine Section and The Department of Medicine, University Hospital, Lund SE-22362, Sweden

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:

  • hematopoietic system;
  • genetic disorders;
  • gene therapy;
  • clinical applications

Summary

In this chapter are examined clinical and preclinical data from the genetic correction of a selection of single-gene disorders that affect the hematopoietic system. Because more than 4,000 diseases are thought to be caused by single-gene defects, this sample represents a minute fraction of available treatment targets. Hematopoietic stem cells are long lived and have the capability to differentiate into every cell lineage of the blood system. The main challenge in the field is therefore to develop strategies to transduce these pluripotent cells. As is shown in this chapter, there are other long-lived cells that are also interesting targets for therapy, for example, T cells.

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