Gene transfer to primary chronic granulomatous disease monocytes

Thrasher, Adrian J. and Casimir, Colin M. and Kinnon, C. and Morgan, G. and Segal, Anthony W. and Levinsky, R. J. (1995) Gene transfer to primary chronic granulomatous disease monocytes. Lancet, 346 (8967). pp. 92-93. ISSN 0140-6736

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For somatic gene therapy to become a realistic therapeutic strategy for 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 defective gene will result in correction of the cellular defect. We used an adenovirus vector expressing p47phox to transduce monocytes from patients with CGD. We showed by nitroblue-tetrazolium staining that NADPH-oxidase activity was restored to these cells. This technique offers a rapid means for molecular diagnosis. In the short term, this approach may have therapeutic potential.

Item Type:Article
Research Areas:School of Science and Technology > Natural Sciences
School of Science and Technology > Natural Sciences > Molecular Biology group
Citations on ISI Web of Science:14
ID Code:3293
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Deposited On:02 Dec 2009 12:02
Last Modified:09 Oct 2014 11:56

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