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  • Title: [Prospects for gene therapy in hematology].
    Author: Moreau-Gaudry F, Mazurier F, Salesse S, Fontanellas A, Verneuil HD.
    Journal: Ann Biol Clin (Paris); 1999; 57(1):43-50. PubMed ID: 9920966.
    Abstract:
    Gene transfer in hematopoietic cells is intended to treat patients with malignant disease and inherited monogenic (hematological, immunological, and metabolic) disorders. Hematopoietic progenitor or stem cells are a favoured target for gene therapy because these cells are easily withdrawn from the patient, expanded and genetically modified ex vivo and then reinjected into the organism. Retroviral vectors allow an efficient transfer of the genes of interest. Transduction of stem cells leads to a stable expression of the transgene for long periods of time. However, we are at the beginning of this new therapeutic application, the technique was being already successful in very few cases. Problems to be solved are mainly in the understanding of the physiology of the hematopoietic stem cell and in the improvement of technical qualities of the vectors for a targeted gene transfer in vivo.
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