{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"submitter":["George BM"],"funding":["Sontag Foundation","Burroughs Wellcome Fund","Medical Research Council","Wellcome Trust","Damon Runyon Cancer Research Foundation","Damon Runyon Cancer Research Foundation (Cancer Research Fund of the Damon Runyon-Walter Winchell Foundation)","Max-Planck-Gesellschaft"],"pubmed_abstract":["Immunotherapies for acute myeloid leukemia (AML) and other cancers are limited by a lack of tumor-specific targets. Here we discover that RNA-binding proteins and glycosylated RNAs (glycoRNAs) form precisely organized nanodomains on cancer cell surfaces. We characterize nucleophosmin (NPM1) as an abundant cell surface protein (csNPM1) on a variety of tumor types. With a focus on AML, we observe csNPM1 on blasts and leukemic stem cells but not on normal hematopoietic stem cells. We develop a monoclonal antibody to target csNPM1, which exhibits robust anti-tumor activity in multiple syngeneic and xenograft models of AML, including patient-derived xenografts, without observable toxicity. We find that csNPM1 is expressed in a mutation-agnostic manner on primary AML cells and may therefore offe"],"journal":["Nature biotechnology"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC7618518"],"repository":["biostudies-literature"],"pubmed_title":["Treatment of acute myeloid leukemia models by targeting a cell surface RNA-binding protein."],"pmcid":["PMC7618518"],"funding_grant_id":["218481/Z/19/Z","RG94424","203151","#74-23","MR/X008371/1","RG83195","RG106133","218481","306752","306752/Z/23/Z"],"pubmed_authors":["Zaro BW","Liu J","Eleftheriou M","de la Rosa J","Yu Q","Yankova E","George BM","Bagri J","Stone RM","Nestola G","Camargo F","Baxter J","Rak J","Volk RF","Evans S","Spiekermann M","Bapcum E","Mockl L","Lebedenko CG","Chai P","Hemberger H","Vassiliou GS","Almahayni K","Russell J","Huntly BJP","Tzelepis K","Damaskou A","Flynn RA","Perr J","Giotopoulos G"],"additional_accession":[]},"is_claimable":false,"name":"Treatment of acute myeloid leukemia models by targeting a cell surface RNA-binding protein.","description":"Immunotherapies for acute myeloid leukemia (AML) and other cancers are limited by a lack of tumor-specific targets. Here we discover that RNA-binding proteins and glycosylated RNAs (glycoRNAs) form precisely organized nanodomains on cancer cell surfaces. We characterize nucleophosmin (NPM1) as an abundant cell surface protein (csNPM1) on a variety of tumor types. With a focus on AML, we observe csNPM1 on blasts and leukemic stem cells but not on normal hematopoietic stem cells. We develop a monoclonal antibody to target csNPM1, which exhibits robust anti-tumor activity in multiple syngeneic and xenograft models of AML, including patient-derived xenografts, without observable toxicity. We find that csNPM1 is expressed in a mutation-agnostic manner on primary AML cells and may therefore offe","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Apr","modification":"2026-07-14T17:28:21.851Z","creation":"2026-06-23T03:09:40.634Z"},"accession":"S-EPMC7618518","cross_references":{"pubmed":["40269321"],"doi":["10.1038/s41587-025-02648-2"]}}