{"entity":{"id":"wt1","kind":"target","name":"WT1","aka":["WT1 transcription factor","Wilms tumor protein","WIT-2","AWT1","NPHS4","WT-1"],"tldr":"WT1 (Wilms tumour protein) is a protein that switches other genes on and off. The public catalogues list it as a drug target, an oncogene driver, a tumour suppressor, a biomarker and a fusion partner, and clinical evidence ties its variants to diagnosis, prognosis or drug response. Tied to Leukaemia, Mesothelioma, Myeloproliferative neoplasms and 5 more.","summary":"Transcription factor that plays an important role in cellular development and cell survival. Recognises and binds to the DNA sequence 5'-GCG(T/G)GGGCG-3'. Regulates the expression of numerous target genes, including EPO.\n\nCIViC holds 12 clinical evidence items and 0 assertions across 3 variants, naming Daunorubicin and Cytarabine. Open Targets scores its association with cancer at 0.90 (direct and indirect evidence; datatypes clinical 0.63, genetic literature 0.90, affected pathway 0.36, literature 1.00, genetic association 0.91, somatic mutation 0.89, animal model 0.68). IntOGen calls it a driver in 4 cohorts (1 activating, 3 loss-of-function), covering Acute Myeloid Leukaemia, Melanoma, Pancreatic Adenocarcinoma. In OnCo, 1 product record names it (INO-5401).","asOf":"2026-09-23","links":[{"label":"HGNC HGNC:12796","url":"https://www.genenames.org/data/gene-symbol-report/#!/hgnc_id/HGNC:12796"},{"label":"UniProt P19544","url":"https://www.uniprot.org/uniprotkb/P19544/entry"},{"label":"NCBI Gene 7490","url":"https://www.ncbi.nlm.nih.gov/gene/7490"},{"label":"Ensembl ENSG00000184937","url":"https://www.ensembl.org/Homo_sapiens/Gene/Summary?g=ENSG00000184937"}],"tags":["cancer-genes-wave"],"related":["civic","open-targets","intogen"],"cancers":["leukaemia","mesothelioma","myeloproliferative-neoplasms","non-hodgkin-lymphoma","sarcoma","pancreatic","wilms-tumor","aml"],"sections":[],"technologies":[],"targets":[],"drugs":["ino-5401"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Written by scripts/fetch-cancer-genes.ts from CIViC, Open Targets, IntOGen, HGNC and UniProt; the function text is UniProt's, condensed and in UK spelling. Roles: Open Targets known-drug datatype score 0.63; IntOGen calls it an activating (Act) driver in 1 cohort; IntOGen calls it a loss-of-function (LoF) driver in 3 cohorts; CIViC holds 12 clinical evidence items on its variants; UniProt disease notes describe a translocation or gene fusion involving the gene. Evidence tier \"clinical-evidence\" is the strongest of those signals.","Prevalence not recorded: none of the sources gives a positivity rate."],"provenance":{"editedBy":"scripts/fetch-cancer-genes.ts (CIViC, Open Targets, IntOGen, HGNC, UniProt)","editedOn":"2026-09-23"},"symbol":"WT1","role":["drug-target","oncogene-driver","tumour-suppressor","biomarker","fusion-partner"],"evidenceTier":"clinical-evidence","sources":[{"label":"HGNC HGNC:12796","url":"https://www.genenames.org/data/gene-symbol-report/#!/hgnc_id/HGNC:12796","note":"approved symbol, name, aliases, locus and cross-references (hgnc_complete_set.txt)"},{"label":"UniProt P19544","url":"https://www.uniprot.org/uniprotkb/P19544/entry","note":"protein name, function text, keywords and locations (REST API)"},{"label":"CIViC gene WT1","url":"https://civicdb.org/features/49","note":"12 evidence items, 0 assertions, 3 variants; diseases: Acute Myeloid Leukaemia (GraphQL API, CC0)"},{"label":"Open Targets ENSG00000184937","url":"https://platform.opentargets.org/target/ENSG00000184937/associations","note":"association with cancer (MONDO_0004992) 0.90; per-cancer scores at or above 0.5: sarcoma 0.59, malignant mesothelioma 0.69, acute myeloid leukaemia 0.65, acute lymphoblastic leukaemia 0.56, non-Hodgkin lymphoma 0.60, myeloproliferative neoplasm 0.68 (GraphQL API, CC0)"},{"label":"IntOGen WT1","url":"https://www.intogen.org/search?gene=WT1","note":"driver in 4 cohorts (Act 1, LoF 3); Compendium_Cancer_Genes.tsv release 20240920, CC0 1.0"}],"specificity":"tumour-associated","distribution":"many-types","specificityNote":"Tumour-associated overexpression: HPA finds the RNA cancer enriched in cancer (Ovary Serous Cystadenocarcinoma (TCGA)) and tissue enhanced in normal endometrium 1, fallopian tube, ovary, smooth muscle, so the tumour and the normal tissue it comes from share the target and the medicine relies on the difference in level. HPA WT1: RNA tissue enhanced (endometrium 1 42 nTPM, fallopian tube 49 nTPM, ovary 28 nTPM); high antibody staining in 5 normal tissues; highest cancer staining glioma (8 of 12 high). Distribution: 7 cancer families in the corpus carry a prevalence row, label threshold or catalogue link for it (Leukaemia, Mesothelioma, Myeloid neoplasms, Lymphoma, Sarcomas (soft tissue, bone, GIST), Pancreatic ductal adenocarcinoma, Childhood cancers (all types)); Open Targets associates it with 7 specific cancer types at or above 0.5 (Wilms tumor 1, malignant mesothelioma, Nephroblastoma, Wilms tumor, WAGR syndrome, acute myeloid leukemia and more). (Rule 7 of scripts/fetch-target-specificity.ts.)","specificitySources":[{"label":"Human Protein Atlas WT1 tissue","url":"https://www.proteinatlas.org/ENSG00000184937-WT1/tissue","note":"RNA tissue and blood lineage specificity, normal tissue antibody staining (version 25.1, CC BY-SA 3.0)"},{"label":"Human Protein Atlas WT1 pathology","url":"https://www.proteinatlas.org/ENSG00000184937-WT1/pathology","note":"patients per staining level per cancer type (version 25.1, CC BY-SA 3.0)"},{"label":"Open Targets ENSG00000184937 associations","url":"https://platform.opentargets.org/target/ENSG00000184937/associations","note":"cancer associations at or above 0.5 (CC0)"}],"hgnc":"HGNC:12796","ensembl":"ENSG00000184937","uniprot":"P19544","entrez":"7490","firstDescribed":1990,"firstDescribedBasis":"sequence","firstDescribedNote":"Earliest sequence paper UniProt cites for the protein: Gessler et al, Nature, 1990, \"Homozygous deletion in Wilms tumours of a zinc-finger gene identified by chromosome jumping\".","firstDescribedSource":"https://pubmed.ncbi.nlm.nih.gov/2154702/","biology":"Transcription factor that plays an important role in cellular development and cell survival. Recognises and binds to the DNA sequence 5'-GCG(T/G)GGGCG-3'. Regulates the expression of numerous target genes, including EPO. Plays an essential role for development of the urogenital system. It has a tumour suppressor as well as an oncogenic role in tumour formation. Function may be isoform-specific: isoforms lacking the KTS motif may act as transcription factors. Location: Nucleus; Nucleus, nucleolus; Cytoplasm; Nucleus speckle (UniProt). Locus 11p13 (HGNC).","whereFound":["Leukaemia: Open Targets association 0.72 with leukaemia (MONDO_0005059)","Mesothelioma: Open Targets association 0.69 with malignant mesothelioma (MONDO_0006292)","Myeloproliferative neoplasms: Open Targets association 0.68 with myeloproliferative neoplasm (MONDO_0020076)","Non-Hodgkin lymphoma: Open Targets association 0.60 with non-Hodgkin lymphoma (MONDO_0018908)","Sarcomas: Open Targets association 0.59 with sarcoma (MONDO_0005089)","Pancreatic ductal adenocarcinoma: IntOGen driver in 1 cohort (PAAD)"],"targetClass":"transcription","prevalence":[]},"route":"/targets/wt1/","neighbours":{"collection":[{"id":"civic","kind":"collection","name":"CIViC","route":"/collections/civic/"},{"id":"intogen","kind":"collection","name":"IntOGen","route":"/collections/intogen/"},{"id":"open-targets","kind":"collection","name":"Open Targets Platform","route":"/collections/open-targets/"}],"cancer":[{"id":"aml","kind":"cancer","name":"Acute myeloid leukaemia","route":"/cancers/aml/"},{"id":"desmoplastic-small-round-cell-tumour","kind":"cancer","name":"Desmoplastic small round cell tumour","route":"/cancers/desmoplastic-small-round-cell-tumour/"},{"id":"leukaemia","kind":"cancer","name":"Leukaemia (all types)","route":"/cancers/leukaemia/"},{"id":"mesothelioma","kind":"cancer","name":"Mesothelioma","route":"/cancers/mesothelioma/"},{"id":"myeloproliferative-neoplasms","kind":"cancer","name":"Myeloproliferative neoplasms (PV, ET, myelofibrosis)","route":"/cancers/myeloproliferative-neoplasms/"},{"id":"non-hodgkin-lymphoma","kind":"cancer","name":"Non-Hodgkin lymphoma (all types)","route":"/cancers/non-hodgkin-lymphoma/"},{"id":"pancreatic","kind":"cancer","name":"Pancreatic ductal adenocarcinoma","route":"/cancers/pancreatic/"},{"id":"sarcoma","kind":"cancer","name":"Sarcomas (soft tissue, bone, GIST)","route":"/cancers/sarcoma/"},{"id":"wilms-tumor","kind":"cancer","name":"Wilms tumour (nephroblastoma)","route":"/cancers/wilms-tumor/"}],"drug":[{"id":"ino-5401","kind":"drug","name":"INO-5401","route":"/drugs/ino-5401/"}]}}