Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/β2-microglobulin–/– repopulating mobilized human peripheral blood CD34+ cells

In previous studies amphotropic MFGS-gp91phox (murine onco-retrovirus vector) was used in a clinical trial of X-linked chronic granulomatous disease (X-CGD) gene therapy to achieve transient correction of oxidase activity in 0.1% of neutrophils. We later showed that transduced CD34+ peripheral blood...

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Veröffentlicht in:	Blood 2003-10, Vol.102 (8), p.2789-2797
Hauptverfasser:	Brenner, Sebastian, Whiting-Theobald, Narda L., Linton, Gilda F., Holmes, Kevin L., Anderson-Cohen, Mindy, Kelly, Patrick F., Vanin, Elio F., Pilon, André M., Bodine, David M., Horwitz, Mitchell E., Malech, Harry L.
Format:	Artikel
Sprache:	eng
Schlagworte:	Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Applied cell therapy and gene therapy Biological and medical sciences Immunodeficiencies Immunodeficiencies. Immunoglobulinopathies Immunopathology Medical sciences Transfusions. Complications. Transfusion reactions. Cell and gene therapy
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