Jpn. J. Infect. Dis., 57, S27-S28, 2004
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Progress Toward Effective Gene Therapy for
Chronic Granulomatous Disease
Harry L. Malech*, Uimook Choi and Sebastian Brenner
Laboratory of Host Defenses, National Institute of Allergy
and Infectious Diseases, National Institutes of Health, Bethesda,
Maryland, USA
*Corresponding author: hmalech@nih.gov
SUMMARY: Previous clinical studies of ex vivo gene therapy for
chronic granulomatous disease (CGD) without marrow conditioning
have resulting in transient correction of the oxidase defect in
over 0.1% of circulation neutrophils. Use of improved RD114 envelope
pseudotyped vectors capable of transducing >95% of CD34+ stem
cells ex vivo, together with non-ablative marrow conditioning
will be incorporated into the next generation of clinical trials
of ex vivo gene therapy for CGD. These maneuvers might result
in clinical benefit to CGD patients from gene therapy.