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Target intelligence / Profile preview
Wilms tumor protein (WT1)
- Target
- WT1
- Molecular classification
- Transcription factor, Kruppel-like zinc finger transcription factor
01
Overview
Wilms tumor protein (WT1) is a transcription factor essential for normal development of the urogenital system, particularly the kidneys and gonads. It regulates genes involved in cell differentiation, proliferation, apoptosis, epithelial/mesenchymal transition, and mRNA splicing. Mutations or deletions of WT1 are associated with several diseases including Wilms' tumor, Denys–Drash syndrome, Frasier syndrome, and WAGR syndrome. WT1 can act both as a tumor suppressor and as an oncogene depending on the cellular context. Overexpression is observed in certain leukemias.
- Other names
- Wilms' tumor protein
02
Mechanism of action
Modulation of WT1 transcriptional activity (e.g., inhibition of its oncogenic function or restoration of its tumor suppressor function).
03
Biological functions
Transcription regulationCell differentiationCell proliferationApoptosisEpithelial/mesenchymal transitionmRNA splicingUrogenital developmentKidney developmentTissue homeostasis
04
Disease associations
Wilms' tumorDenys–Drash syndromeFrasier syndromeWAGR syndromeCancer
05
Safety considerations
Potential for off-target effects due to broad transcriptional regulationContext-dependent tumor suppressor vs. oncogenic rolesDevelopmental toxicity due to role in organogenesis
06
Biomarkers
WT1 expression levels in leukemic cellsWT1 expression levels in tumor tissuesWT1 mutations in Wilms' tumorWT1 mutations in Denys–Drash syndromeWT1 mutations in Frasier syndrome
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