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Division of Immunology/Hematology/Oncology, University-Children's Hospital Zurich, CH-8032 Zurich, Switzerland
Gene therapy of hematopoietic stem cells (HSC) is limited by low frequency of the target cells, their quiescent nature, poor engraftment of treated HSC, and lack of a selective growth advantage of genetically modified cells. Lentiviral vectors combined with positive selection strategies using conditional cell-growth switches should allow for improvement.
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