Abstract
X-linked severe combined immunodeficiency (X-SCID), a fatal disorder caused by mutations in the IL2R|[gamma]| gene, has been treated by retroviral vector (RV) mediated gene replacement in hematopoietic stem cells (HSC). A crucial factor for the success of this approach was the selective growth advantage of gene-modified cells, which allowed lymphoid repopulation from a small number of transduced HSC. Unfortunately, leukemia developed in a fraction of treated patients and its origin was linked to RV insertional mutagenesis.
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