Target intelligence / Profile preview

Wilms' Tumor 1 Protein (WT1)

Target
WT1
Molecular classification
Transcription factor, Zinc finger protein, RNA-binding protein
01

Overview

Wilms' tumor 1 protein (WT1) is a zinc finger transcription factor that plays a crucial role in urogenital system development. It functions as both a tumor suppressor and an oncogene, depending on the cellular context. Mutations or altered expression are implicated in several cancers, including Wilms’ tumor and acute myeloid leukemia. WT1 regulates gene expression and RNA metabolism, influencing cell growth, differentiation, apoptosis, and organ development. It is also a diagnostic marker for certain tumors.

Other names
Wilms tumor proteinWT1 proteinNephroblastomaWTI
02

Mechanism of action

Modulation of WT1 expression or activity; inhibition of WT1 function; immunotherapy targeting WT1

03

Biological functions

Transcriptional regulationRNA bindingCell growthDifferentiationApoptosisOrgan developmentEpigenetic regulation
04

Disease associations

CancerWilms' tumorAcute myeloid leukemia (AML)Denys–Drash syndromeMesotheliomaOvarian serous carcinomaHernias
05

Safety considerations

Potential for off-target effects due to widespread expressionComplex and context-dependent function makes therapeutic targeting challenging
06

Biomarkers

Minimal residual disease monitoring in AMLImmunohistochemical detection in mesothelioma and ovarian carcinomaWT1 expression levels in AML

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