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• W2006029795 abstract "Transfer of the gene coding for glucocerebrosidase (GC) via a retroviral vector (MFG-GC) to haematopoietic progenitors results in engraftment and life-long expression of the human protein at high levels in transplanted mice. Studies of human CD34 cells were carried out to evaluate their potential use in a gene therapy approach to Gaucher's disease. High transduction efficiency and correction of the enzyme deficiency was possible in CD34 cells obtained from patients with Gaucher's disease. Based on these results, a clinical trial of gene therapy was designed and initiated. Preliminary results of this study indicate the persistence or engraftment of genetically corrected cells in the transplanted patients." @default.
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• W2006029795 date "1997-12-01" @default.
• W2006029795 modified "2023-09-27" @default.
• W2006029795 title "9 Gaucher's disease: studies of gene transfer to haematopoietic cells" @default.
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• W2006029795 doi "https://doi.org/10.1016/s0950-3536(97)80039-x" @default.
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