Scientists from Hopital Necker Enfants Malade (Paris, France) have reportedly confirmed that the gene therapy they used to treat a group of young boys with severe combined immunodeficiency disease (SCID) caused a leukemia-like disease in two of them.There had been a general consensus within the field that insertional mutagenesis was to blame - the therapeutic gene delivered in a retrovirus vector inserted near and activated a gene in the chromosome. Salima Hacien-Bay-Abina, a researcher at the hospital, reported at a meeting of the European Society of Human Genetics that the activated gene has been identified. The therapeutic SCID gene landed inside the cancerpromoting gene LMO-2 in one of the boys and landed near this gene in the other child.
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