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Target intelligence / Profile preview
Wilms Tumor Protein 1 (WT1)
- Target
- WT1
- Molecular classification
- Transcription factor, Zinc finger protein, RNA-binding protein
01
Overview
Wilms tumor protein 1 (WT1) is a zinc finger transcription factor crucial for urogenital system development and normal embryonic development. It functions as both a tumor suppressor and an oncogene depending on the cellular context. WT1 regulates genes involved in cell growth, differentiation, apoptosis, and RNA metabolism. Aberrant expression or mutations are implicated in Wilms tumor, Denys–Drash syndrome, and acute myeloid leukemia (AML). WT1 is a significant biomarker for diagnosis and monitoring of AML, and a target for cancer immunotherapies due to its high immunogenicity.
- Other names
- Wilms Tumor 1WT1 proteinWT1 transcription factor
02
Mechanism of action
WT1 is a target for immunotherapies, where the mechanism of action involves stimulating an immune response (e.g., cytotoxic T lymphocytes) to target and kill WT1-expressing cancer cells.
03
Biological functions
OrganogenesisTranscriptional regulationCell growthCell differentiationApoptosisRNA metabolismEpigenetic regulation
04
Disease associations
CancerWilms tumorAcute Myeloid Leukemia (AML)Denys–Drash syndrome
05
Safety considerations
Off-target effects of immunotherapiesPotential for autoimmunity due to WT1 expression in normal tissues
06
Biomarkers
Minimal Residual Disease (MRD) in AMLRelapse detection in AMLImmunohistochemical marker for mesothelioma and ovarian serous carcinoma
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