{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Werner J"],"funding":["NCI NIH HHS"],"pagination":["3804"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC12019388"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["16(1)"],"pubmed_abstract":["Juvenile myelomonocytic leukemia (JMML) is a myeloproliferative disorder that predominantly affects infants and young children. Hematopoietic stem cell transplantation (HSCT) is standard of care, but post-HSCT relapse is common, highlighting the need for innovative therapies. While adoptive immunotherapy with chimeric antigen receptor (CAR) T cells has improved outcomes for patients with advanced lymphoid malignancies, it has not been comprehensively evaluated in JMML. In the present study, we use bulk and single-cell RNA sequencing, mass spectrometry, and flow cytometry to identify overexpression of CLL-1 (encoded by CLEC12A) on the cell surface of cells from patients with JMML. We develop immunotherapy with CLL-1 CAR T cells (CLL1CART) for preclinical testing and report in vitro and in vivo anti-leukemia activity. Notably, CLL1CART reduce the number of leukemic stem cells and serial transplantability in vivo. These preclinical data support the development and clinical investigation of CLL-1-targeting immunotherapy in children with relapsed/refractory JMML."],"journal":["Nature communications"],"pubmed_title":["Cellular immunotherapy targeting CLL-1 for juvenile myelomonocytic leukemia."],"pmcid":["PMC12019388"],"funding_grant_id":["U54 CA196519","R37 CA266550","P30 CA082103","R50 CA274213"],"pubmed_authors":["Tasian SK","Loh ML","Barpanda A","Morales C","Xirenayi S","Patino-Escobar B","Temple WC","Shin H","Stieglitz E","Chaudhuri S","Mandal K","Wiita AP","Zhang C","Braun B","Yousuf K","Bachl S","Werner J","Abelson S","Meshinchi S","Meyer J","Dardis JK","Rivera J","Bhatnagar S","Izgutdina A","Lee AG","Ramos E"],"additional_accession":[]},"is_claimable":false,"name":"Cellular immunotherapy targeting CLL-1 for juvenile myelomonocytic leukemia.","description":"Juvenile myelomonocytic leukemia (JMML) is a myeloproliferative disorder that predominantly affects infants and young children. Hematopoietic stem cell transplantation (HSCT) is standard of care, but post-HSCT relapse is common, highlighting the need for innovative therapies. While adoptive immunotherapy with chimeric antigen receptor (CAR) T cells has improved outcomes for patients with advanced lymphoid malignancies, it has not been comprehensively evaluated in JMML. In the present study, we use bulk and single-cell RNA sequencing, mass spectrometry, and flow cytometry to identify overexpression of CLL-1 (encoded by CLEC12A) on the cell surface of cells from patients with JMML. We develop immunotherapy with CLL-1 CAR T cells (CLL1CART) for preclinical testing and report in vitro and in vivo anti-leukemia activity. Notably, CLL1CART reduce the number of leukemic stem cells and serial transplantability in vivo. These preclinical data support the development and clinical investigation of CLL-1-targeting immunotherapy in children with relapsed/refractory JMML.","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Apr","modification":"2026-06-02T19:06:02.158Z","creation":"2026-04-19T03:14:02.238Z"},"accession":"S-EPMC12019388","cross_references":{"pubmed":["40268927"],"doi":["10.1038/s41467-025-59040-6"]}}