Chapter 15. Gene Therapy for Hemophilia
- Peter J. Quesenberry2,
- Gary S. Stein2,
- Bernard G. Forget3,
- Sherman M. Weissman3
Published Online: 13 MAY 2002
DOI: 10.1002/0471223956.ch15
Copyright © 1998 by Wiley-Liss, Inc.
Book Title
Stem Cell Biology and Gene Therapy
Additional Information
How to Cite
High, K. A. (2002) Gene Therapy for Hemophilia, 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.ch15
Editor Information
- 2
University of Massachusetts, Worcester, Massachusetts
- 3
Yale University School of Medicine, New Haven, Connecticut
Publication History
- Published Online: 13 MAY 2002
- Published Print: 27 AUG 1998
ISBN Information
Print ISBN: 9780471146568
Online ISBN: 9780471223955
- Summary
- Chapter
Keywords:
- hemophilia;
- gene therapy;
- transferred genes;
- retroviruses;
- F.IX gene transfer;
- adenoviral vectors;
- adeno-associated virus (AAV) vectors;
- F.VIII gene transfer;
- nonviral approaches
Summary
Hemophilia is the X-linked bleeding diathesis that results from a deficiency of functional Factor VIII (hemophilia A) or Factor IX (hemophilia B). The disease occurs worldwide and affects approximately 1 in 10,000 male births. Hemophilia A is approximately five times as common as hemophilia B; it has been shown for X-linked diseases that the frequency of disease bears a direct relationship to the size of the affected gene. This would appear to hold true for the hemophilias, where the Factor VIII (F.VIII) gene is 186 kb in length and the Factor IX (F.IX) gene is 35 kb. Gene therapy for hemophilia is the subject of this chapter.