Abstract:Three and five years after an otherwise successful gene therapy trial for X1-SCID (gamma c deficiency) three out of 11 patients developed T cell leukemia due to insertional activation of the proto-oncogene LMO2 after retroviral mediated gamma c transfer into autologous CD34+ cells. In one of the patient’s T-cells SCL(TAL1) was expressed in addition to LMO2 because of a SIL-SCL fusion, where the deletion of the SIL gene brings SCL(TAL1) under the control of the constitutively active SIL promoter. LMO2 and SCL(T… Show more
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