Wilms' tumor 1 gene in hematopoietic malignancies: clinical implications and future directions

Leuk Lymphoma. 2020 Sep;61(9):2059-2067. doi: 10.1080/10428194.2020.1762884. Epub 2020 May 13.


The Wilms' tumor 1 (WT1) gene is an important regulatory molecule that plays a vital role in cell growth and development. Initially, knowledge of WT1 was mostly limited to Wilms' tumor. Over the past years, numerous studies have shown that WT1 is aberrant expressed or mutated in hematopoietic malignancies, including acute leukemia (AL), myelodysplastic syndrome (MDS) and chronic myelogenous leukemia (CML). Currently, many studies focus on exploring the role of WT1 in hematopoietic malignancies. Such studies improve the understanding of hematopoietic malignancies, and the collection of data about WT1 expression or mutation in hematopoietic malignancies over the past years can facilitate the risk stratification of hematopoietic malignancies. In this review, we highlight the important role of WT1 in hematopoietic malignancies, discuss its potential clinical applications as a minimal residual disease (MRD) and prognostic biomarker, and evaluate the possible therapy target of WT1 in hematopoietic malignancies.

Keywords: AML; Hematopoietic malignancies; MDS; MRD; WT1.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Hematologic Neoplasms* / diagnosis
  • Hematologic Neoplasms* / genetics
  • Humans
  • Leukemia, Myeloid, Acute*
  • Myelodysplastic Syndromes*
  • Neoplasm, Residual / genetics
  • WT1 Proteins / genetics
  • Wilms Tumor*


  • WT1 Proteins