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pubmed-article:7584079pubmed:abstractTextOne promising strategy for gene therapy of Gaucher disease involves ex vivo retroviral transduction of autologous hematopoietic stem cells. Studies in small animals have demonstrated that this approach provides a life-long supply of the glucocerebrosidase (GC) enzyme. Human application has developed to the stage of a clinical trial. In this study, we describe development of a high titer amphotropic producer line for the vector, MFG-GC, and explore transduction of CD34+ cells from various human sources. Higher than three times the normal levels of glucocerebrosidase activity in non-transduced cells were achieved following transduction of CD34+ cells obtained from bone marrow or cord blood from normal donors. The improvement in enzyme activity in Gaucher marrow was about 40-fold above deficient levels. We examined the timing and stepwise effect of multiple rounds of infection and evaluated post-infection expansion of cells in two different cytokine mixtures. Transduction efficiency was determined using immunocytochemistry and Southern blot hybridization.lld:pubmed
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pubmed-article:7584079pubmed:pagination176-84lld:pubmed
pubmed-article:7584079pubmed:dateRevised2007-11-14lld:pubmed
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pubmed-article:7584079pubmed:articleTitleTransduction of CD34+ enriched cord blood and Gaucher bone marrow cells by a retroviral vector carrying the glucocerebrosidase gene.lld:pubmed
pubmed-article:7584079pubmed:affiliationDepartment of Human Genetics, Graduate School of Public Health, University of Pittsburgh, USA.lld:pubmed
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