The possible role and application of WT1 in human leukemia

WT1, a tumor suppressor gene responsible for the development of childhood k idney tumors, is now also thought to be involved in the occurrence of human leukemia. First, evidence has shown that WT1 functions during hematopoiesi s and regulates the proliferation and differentiation of blood cells. Secon d, specific expression patterns of this gene correlate with the malignant p henotype of leukemia compared with the physiological situation. Third, muta tions of WT1 can be detected, though not frequently, in human leukemia but not in normal hematopoietic cells. Thus, a possible role of WT1 in human le ukemogenesis has been proposed. Because the expression of this gene is rela tively high during the so-called myelodysplastic stages and in all subtypes of human leukemia compared with normal blood cells, the notion has been ra ised that WT1 can be used as a 'panleukemic marker' for the diagnosis of le ukemia at the molecular level. The expression level of WT1 may have signifi cance in predicting prognosis and monitoring relapse. Moreover, with a deep er understanding of its role in leukemogenesis, WT1 may serve as a target m olecule in the strategy of gene therapy for leukemia. (C) 2001 The Japanese Society of Hematology.