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  • Title: [Gene therapy for severe combined immunodeficiency].
    Author: Petersen LB, Jensen TG.
    Journal: Ugeskr Laeger; 2010 Jun 07; 172(23):1756-8. PubMed ID: 20534204.
    Abstract:
    New results with gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency are promising. We review a clinical project in which ten children were treated with gene modified autologous haematopoietic stem cells. After treatment, eight patients were able to do without enzyme-replacement therapy, and nine patients showed improved immune function and sustained low concentration of toxic metabolites. No clonal outgrow was observed indicating a limited risk for future malignant development. Despite these promising results, the safety of gene therapy can still be improved.
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