| 摘要 |
The invention relates to the use of genetically modified, very early haematopoietic and mesenchymal stem cells (negative for the expression of th e surface molecule CD34) in the individual gene therapy of mono- or oligogenet ic diseases or in cell therapy. Autologous CD34-negative adherently growing ste m cell cultures from the peripheral blood of the patient are applied and efficiently transfected or infected with genetic constructs. The gene produc ts of these genes should substitute defective or absent proteins or factors in the patient organism in the long term. After expansion, the autologous stem cells can also be used for cell therapy (organ replacement therapy).
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