Gene therapy for inherited immunodeficiencies.

Abstract

Extensive research into gene therapy technologies has produced few clinically relevant results. Advances in the understanding of the genetics of inherited immunologic diseases, gene transfer methodologies, and stem cell manipulation have all contributed to successes in treating X-linked severe combined immunodeficiency and adenosine deaminase deficiency. This review examines the progress and the problems that have arisen, and discusses the improvement and future of gene therapy for primary immunodeficiencies.

• Authors: Steven Howe, Adrian J Thrasher
• Journal: Current hematology reports (Curr Hematol Rep) Vol. 2 Issue 4 Pg. 328-34 (Jul 2003) ISSN: 1540-3408 [Print] United States
• PMID: 12901330 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)

Chemical References

• Adenosine Deaminase

Topics

• Adenosine Deaminase (deficiency)
• Genetic Therapy (methods)
• Hematopoietic Stem Cells
• Humans
• Immunologic Deficiency Syndromes (pathology, therapy)
• Leukopoiesis
• Severe Combined Immunodeficiency (pathology, therapy)