Abstract
Chronic granulomatous disease (CGD) is a primary immunodeficiency disorder which results from absence or malfunction of the respiratory burst oxidase normally expressed in neutrophils and other phagocytic leukocytes. Two-thirds of the patients are males hemizygous for mutations in the X-linked gene coding for gp91-phox. As a therapeutic approach towards the X-linked form of CGD bicistronic retroviral vectors containing the gp91-phox gene and a selectable marker gene were constructed. The ability of these vectors to restore NADPH oxidase activity was tested in a human myeloid leukemic cell line that is defective in superoxide production, as well as in primary CD34+ cells obtained from X-CGD patients. Under optimal conditions 80% of the CD34+ cells derived from bone marrow of one X-CGD patient were transduced. The level of superoxide production, in phagocytes derived from transduced cells was 68.9% of normal levels. Considering that low levels of superoxide generating activity are sufficient for normal host defense, the present experiments provide the basis for the development of a gene replacement therapy for the X-linked form of CGD. Bone Marrow Transplantation (2000) 25, Suppl. 2, S99–S104.
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Grez, M., Becker, S., Saulnier, S. et al. Gene therapy of chronic granulomatous disease. Bone Marrow Transplant 25 (Suppl 2), S99–S104 (2000). https://doi.org/10.1038/sj.bmt.1702365
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DOI: https://doi.org/10.1038/sj.bmt.1702365
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