Xaver hutter biography of albert

To the Editor:

Ten pediatric patients with severe Wiskott–Aldrich syndrome (WAS) have received autologous bone marrow transplantation of gene-corrected CD34+ cells within a hematopoietic stem cell gene therapy (GT) trial [1]. Following GT, nine out of ten treated patients had nearly complete remission of their clinical disease and showed sustained expression of WAS protein in myeloid and lymphoid cells. However, 1–5 years later, they developed T-cell acute lymphoblastic leukemia (T-ALL) and/or acute myeloid leukemia. The clinical course of these patients and characterization of the retroviral integration sites within the leukemic clones have been reported [2]. All six T-ALL clones harbored retroviral vector integrations within or close to the LMO2 gene locus and one to eight additional integration sites, including integrations upstream of T-ALL proto-oncogenes TAL1 (WAS5 and WAS7) and LYL1 (WAS8 and WAS10). LMO2, a transcriptional regulator of hematopoiesis, is continuously d

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