title: Progress toward effective gene therapy for chronic granulomatous disease.
abstract: 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.
authors.affiliation: Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA. hmalech@nih.gov
authors.forename: Harry L
authors.initials: HL
authors.lastname: Malech
authors.forename: Uimook
authors.initials: U
authors.lastname: Choi
authors.forename: Sebastian
authors.initials: S
authors.lastname: Brenner
mesh_terms: D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006105:Granulomatous Disease, Chronic / Q000235:genetics* / Q000628:therapy*; D006801:Humans; D008562:Membrane Glycoproteins / Q000235:genetics; D000074662:NADPH Oxidase 2; D019255:NADPH Oxidases / Q000235:genetics; D010750:Phosphoproteins / Q000235:genetics
chemical_list: D008562:Membrane Glycoproteins; D010750:Phosphoproteins; C056558:CYBB protein, human; D000074662:NADPH Oxidase 2; D019255:NADPH Oxidases; C060505:neutrophil cytosolic factor 1