Activation of the T-Cell Oncogene<i>LMO2</i>after Gene Therapy for X-Linked Severe Combined Immunodeficiency

Abstract excerpt

Gene therapy, in which a retrovirus was used to carry the relevant gene (IL2Rγc) into a patient's hematopoietic stem cells, was used to treat 10 boys with X-linked severe combined immunodeficiency (SCID). A syndrome resembling T-cell leukemia developed in two of the boys. In both boys — and a third in whom leukemia has not developed — the retrovirus integrated into the same genomic site, the locus of LMO2, which...

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