Journal
MOLECULAR THERAPY
Volume 19, Issue 1, Pages 28-35Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/mt.2010.232
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Funding
- Bundesministerium fur Bildung und Forschung [01GU0507, TP6b, 01GU0811, TP2b]
- Chronic Granulomatous Disorder Research Trust, London [J4G/04B/GT]
- EU
- Deutsche Forschungsgemeinschaft [1230]
- NIH [P01 HL53586]
- Wellcome Trust
- Department of Health [HTH/011/025/004]
- Bundesministerium fur Gesundheit
- Hessisches Ministerium fur Wissenschaft und Kunst
- Georg-Speyer-Haus [T300131]
- Great Ormond Street Hospital Childrens Charity [V1223] Funding Source: researchfish
- NATIONAL HEART, LUNG, AND BLOOD INSTITUTE [P01HL053586] Funding Source: NIH RePORTER
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The potential of gene therapy as a curative treatment for monogenetic disorders has been clearly demonstrated in a series of recent Phase I/II clinical trials. Among primary immunodeficiencies, gene transfer into hematopoietic stem (HSC)/progenitor cells has resulted in the long-term correction of immune and metabolic defects in treated patients. In most cases, successes were augmented by a recognized biological selection for successfully treated cells in vivo, perhaps even to some extent at the HSC level. In contrast, similar achievements have not turned into reality for immunodeficiencies in which gene-transduced cells lack selective advantages in vivo. This is the case for chronic granulomatous disease (CGD), a primary immunodeficiency, characterized by deficient antimicrobial activity in phagocytic cells. Several attempts to correct CGD by gene transfer in combination with bone marrow conditioning have resulted in low-level long-term engraftment and transient clinical benefits despite high levels of gene marking and high numbers of reinfused cells. This review summarizes the data from clinical trials for CGD and provides some insights into treatment options that may lead to a successful application of gene therapy for CGD.
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