Gene therapy for chronic granulomatous disease

Chronic granuloniatous disease (CGD) is a rare inherited imnunodeficiency characterized by recurrent, often life threatening bacterial and fungal infections due to a functional defect in the microbial-killing activity of phagocytic neutrophils. If regular care and conventional therapy fail, tile dis...

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Bibliographic Details
Published in:	Current opinion in molecular therapeutics Vol. 8; no. 5; p. 415
Main Authors:	Stein, Stefan, Siler, Ulrich, Ott, Marion G, Seger, Reinhard, Grez, Maunel
Format:	Journal Article
Language:	English
Published:	England 01-10-2006
Subjects:	Bone Marrow Transplantation - methods Genetic Therapy - methods Genetic Vectors - genetics Granulomatous Disease, Chronic - therapy Humans Retroviridae - genetics
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Summary:	Chronic granuloniatous disease (CGD) is a rare inherited imnunodeficiency characterized by recurrent, often life threatening bacterial and fungal infections due to a functional defect in the microbial-killing activity of phagocytic neutrophils. If regular care and conventional therapy fail, tile disease can be cured by bone marrow transplantation. This treatment is, however, only available to patients with human leukocyte antigen-identical sibling or matched unrelated donors. One therapeutic option for patients lacking suitable donors is the genetic modification of autologous hematopoietic stem cells. This review discusses the developments that have led to the realization of a successful gene therapy protocol for the correction of CGD.
ISSN:	1464-8431