PMID: 7541496Jul 8, 1995Paper

Gene transfer to primary chronic granulomatous disease monocytes

Lancet
A J ThrasherR J Levinsky

Abstract

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.

References

Sep 23, 1982·The New England Journal of Medicine·E S Buescher, J I Gallin
Oct 21, 1994·Biochimica Et Biophysica Acta·A J ThrasherA W Segal

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Citations

Dec 21, 2006·Molecular Therapy : the Journal of the American Society of Gene Therapy·Ronald R NepomucenoGlen R Nemerow
Aug 23, 2005·Medical Mycology·N G AlmyroudisB H Segal
Aug 24, 1999·Journal of Interferon & Cytokine Research : the Official Journal of the International Society for Interferon and Cytokine Research·D ConteM T Bardella
Aug 31, 2002·Journal of Hematotherapy & Stem Cell Research·Marion-Gabriele OttManuel Grez
Jul 25, 2000·Postgraduate Medical Journal·D G James
May 30, 2008·Seminars in Immunopathology·Marie José Stasia, Xing Jun Li
Jul 22, 2008·La Revue de médecine interne·M J StasiaI Durieu
Dec 29, 2000·Pediatric Clinics of North America·B H Segal, S M Holland
Jan 24, 2007·Physiological Reviews·Karen Bedard, Karl-Heinz Krause

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