GENE-TRANSFER TO PRIMARY CHRONIC GRANULOMATOUS-DISEASE MONOCYTES

For somatic gene therapy to become a realistic therapeutic strategy fo r chronic granulomatous disease (CGD),we have to be able to assign the molecular lesion to a specific component of the NADPH oxidase and to confirm that transfer of a functional copy of the corresponding defect ive gene will result in correction of the cellular defect. We used an adenovirus Vector expressing p47phox to transduce monocytes from patie nts with CGD. We showed by nitroblue-tetrazolium staining that NADPH-o xidase activity was restored to these cells. This technique offers a r apid means for molecular diagnosis. in the short term, this approach m ay have therapeutic potential.