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Target intelligence / Profile preview
Wilms' Tumor Protein 1 (WT1)
- Target
- WT1
- Molecular classification
- Transcription factor
01
Overview
Wilms' tumor protein, commonly referred to as WT1, is a transcription factor encoded by the WT1 gene. It plays a critical role in the development of the urogenital system and is essential for normal kidney formation. Mutations or deletions in this gene are associated with Wilms' tumor and other developmental syndromes. WT1 acts as a transcription factor regulating genes involved in cell differentiation, epithelial/mesenchymal transition control, cell proliferation, and apoptosis. It has both tumor suppressor and oncogenic roles depending on the cellular context.
- Other names
- Wilms Tumor 1WT-1
02
Mechanism of action
Currently, there are no drugs directly targeting WT1. Research focuses on inhibiting WT1 expression or function indirectly through other pathways.
03
Biological functions
Transcriptional regulationCell differentiationEpithelial/mesenchymal transition controlCell proliferationApoptosisKidney developmentUrogenital developmentHematopoiesis
04
Disease associations
Wilms’ tumorDenys–Drash syndromeFrasier syndromeWAGR syndromeCancer
05
Safety considerations
Potential for off-target effects due to broad expression during development and in certain adult tissues.Complex role in both tumor suppression and oncogenesis makes therapeutic targeting challenging.
06
Biomarkers
WT1 expression in tumor tissueWT1 mutations
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