Chapter 11. Ribozyme Gene Therapy Targeting Stem Cells for Human Immunodeficiency Virus Infection
- Peter J. Quesenberry3,
- Gary S. Stein3,
- Bernard G. Forget4,
- Sherman M. Weissman4
Published Online: 13 MAY 2002
DOI: 10.1002/0471223956.ch11
Copyright © 1998 by Wiley-Liss, Inc.
Book Title
Stem Cell Biology and Gene Therapy
Additional Information
How to Cite
Ho, A. D., Law, P., Li, X. and Wong-Staal, F. (2002) Ribozyme Gene Therapy Targeting Stem Cells for Human Immunodeficiency Virus Infection, 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.ch11
Editor Information
- 3
University of Massachusetts, Worcester, Massachusetts
- 4
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:
- HIV infection;
- hematopoiesis;
- HIV life cycle;
- gene therapy;
- RNA decays;
- antisense RNA;
- ribozymes;
- hematopoietic stem cells;
- pluripotent stem cells;
- lineage-committed progenitor cells;
- stem cell infectability;
- stem cell mobilization
Summary
Gene therapy, although still in its infancy, promises to be an exciting new treatment regimen. Its scope has greatly expanded beyond its original conception as a gene replacement strategy for hereditary disorders and now embodies disease targets that include chronic infectious diseases, cancer, and degenerative disorders. For HIV infection, many different approaches using gene transfer to either limit virus replication or stimulate host immunity are in preclinical development, and a number of clinical investigations are currently underway. The antiviral approach has also been referred to as intracellular immunization, as the goal is to render cells genetically resistant to infection. This chapter is not meant to provide an exhaustive and comprehensive review of the entire field, but focuses on the use of hemopoietic stem cells as vehicles for antiviral gene therapy for HIV infection.