{"entity":{"id":"ppat","kind":"target","name":"PPAT","aka":["phosphoribosyl pyrophosphate amidotransferase","Amidophosphoribosyltransferase"],"tldr":"PPAT (Amidophosphoribosyltransferase) is an enzyme. The public catalogues list it as a drug target, and an approved or late-stage drug is recorded against it. Tied to Leukaemia, Non-Hodgkin lymphoma and Acute lymphoblastic leukaemia.","summary":"Catalyses the formation of phosphoribosylamine from phosphoribosylpyrophosphate (PRPP) and glutamine.\n\nOpen Targets scores its association with cancer at 0.58 (direct and indirect evidence; datatypes literature 0.89, animal model 0.56, genetic association 0.60, clinical 0.66).","asOf":"2026-09-23","links":[{"label":"HGNC HGNC:9238","url":"https://www.genenames.org/data/gene-symbol-report/#!/hgnc_id/HGNC:9238"},{"label":"UniProt Q06203","url":"https://www.uniprot.org/uniprotkb/Q06203/entry"},{"label":"NCBI Gene 5471","url":"https://www.ncbi.nlm.nih.gov/gene/5471"},{"label":"Ensembl ENSG00000128059","url":"https://www.ensembl.org/Homo_sapiens/Gene/Summary?g=ENSG00000128059"}],"tags":["cancer-genes-wave"],"related":["open-targets"],"cancers":["leukaemia","non-hodgkin-lymphoma","all-leukemia"],"sections":[],"technologies":[],"targets":[],"drugs":[],"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 0.98. Evidence tier \"approved-drug\" 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":"PPAT","role":["drug-target"],"evidenceTier":"approved-drug","sources":[{"label":"HGNC HGNC:9238","url":"https://www.genenames.org/data/gene-symbol-report/#!/hgnc_id/HGNC:9238","note":"approved symbol, name, aliases, locus and cross-references (hgnc_complete_set.txt)"},{"label":"UniProt Q06203","url":"https://www.uniprot.org/uniprotkb/Q06203/entry","note":"protein name, function text, keywords and locations (REST API)"},{"label":"Open Targets ENSG00000128059","url":"https://platform.opentargets.org/target/ENSG00000128059/associations","note":"association with cancer (MONDO_0004992) 0.58; per-cancer scores at or above 0.5: acute lymphoblastic leukaemia 0.59, non-Hodgkin lymphoma 0.59, leukaemia 0.60 (GraphQL API, CC0)"}],"specificity":"broadly-expressed","distribution":"few-types","specificityNote":"Broadly expressed or essential: HPA lists PPAT among essential proteins and finds the RNA at low tissue specificity; a medicine acting on the wild-type protein would expose normal tissue too. HPA PPAT: RNA low tissue specificity; high antibody staining in 1 normal tissue. Distribution: 2 cancer families in the corpus carry a prevalence row, label threshold or catalogue link for it (Leukaemia, Lymphoma); Open Targets associates it with 1 specific cancer type at or above 0.5 (acute lymphoblastic leukemia). (Rule 7 of scripts/fetch-target-specificity.ts.)","specificitySources":[{"label":"Human Protein Atlas PPAT tissue","url":"https://www.proteinatlas.org/ENSG00000128059-PPAT/tissue","note":"RNA tissue and blood lineage specificity, normal tissue antibody staining (version 25.1, CC BY-SA 3.0)"},{"label":"Open Targets ENSG00000128059 associations","url":"https://platform.opentargets.org/target/ENSG00000128059/associations","note":"cancer associations at or above 0.5 (CC0)"}],"hgnc":"HGNC:9238","ensembl":"ENSG00000128059","uniprot":"Q06203","entrez":"5471","firstDescribed":1993,"firstDescribedBasis":"sequence","firstDescribedNote":"Earliest sequence paper UniProt cites for the protein: Iwahana et al, Biochem. Biophys. Res. Commun, 1993, \"Molecular cloning of human amidophosphoribosyltransferase\".","firstDescribedSource":"https://pubmed.ncbi.nlm.nih.gov/8380692/","biology":"Catalyses the formation of phosphoribosylamine from phosphoribosylpyrophosphate (PRPP) and glutamine. Locus 4q12 (HGNC).","whereFound":["Leukaemia: Open Targets association 0.60 with leukaemia (MONDO_0005059)","Non-Hodgkin lymphoma: Open Targets association 0.59 with non-Hodgkin lymphoma (MONDO_0018908)","Acute lymphoblastic leukaemia: Open Targets association 0.59 with acute lymphoblastic leukaemia (MONDO_0004967)"],"targetClass":"enzyme","prevalence":[]},"route":"/targets/ppat/","neighbours":{"collection":[{"id":"open-targets","kind":"collection","name":"Open Targets Platform","route":"/collections/open-targets/"}],"cancer":[{"id":"all-leukemia","kind":"cancer","name":"Acute lymphoblastic leukaemia","route":"/cancers/all-leukemia/"},{"id":"leukaemia","kind":"cancer","name":"Leukaemia (all types)","route":"/cancers/leukaemia/"},{"id":"non-hodgkin-lymphoma","kind":"cancer","name":"Non-Hodgkin lymphoma (all types)","route":"/cancers/non-hodgkin-lymphoma/"}]}}