iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemia

Abstract CD226 plays a vital role in NK cell cytotoxicity, interacting with its ligands on tumor targets. Acute myeloid leukemia (AML) cells have developed mechanisms to escape NK cell cytotoxicity, including inducing downregulation of CD226 on NK cells. Induced pluripotent stem cell -derived NK (iP...

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Main Authors: Runze Cai, Binyan Lu, Xiangyu Zhao, Shixin Zhou, Yang Li
Format: Article
Language:English
Published: BMC 2025-07-01
Series:Experimental Hematology & Oncology
Subjects:
Online Access:https://doi.org/10.1186/s40164-025-00686-9
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author Runze Cai
Binyan Lu
Xiangyu Zhao
Shixin Zhou
Yang Li
author_facet Runze Cai
Binyan Lu
Xiangyu Zhao
Shixin Zhou
Yang Li
author_sort Runze Cai
collection DOAJ
description Abstract CD226 plays a vital role in NK cell cytotoxicity, interacting with its ligands on tumor targets. Acute myeloid leukemia (AML) cells have developed mechanisms to escape NK cell cytotoxicity, including inducing downregulation of CD226 on NK cells. Induced pluripotent stem cell -derived NK (iPSC-NK) cells offer an important source of standardized off-the-shelf NK cell therapy to treat AML patients. In this study, we engineered iPSC-NK cells with CD226 to assess the ability of killing AML cells. iPSC-NK cells engineered with CD226 have a typical NK cell phenotype and demonstrate improved anti-AML activity and multiple cytokines releasing at low effector-to-target ratios. Transcriptomic analysis revealed upregulation of immune effector function pathways associated with cytotoxicity and immune activation in CD226-overexpression iPSC-NK cells. In an AML xenograft model, mice treated with CD226 overexpression iPSC-NK cells exhibited significantly reduced leukemia burden, prolonged survival, decreased systemic inflammation compared to those treated with Control iPSC-NK cells. Overall, our study provided evidence that iPSC derived-NK cells engineered with CD226 represent a promising candidate for off-the-shelf immunotherapy, particularly in AML and other CD226 ligand-expressing malignancies.
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institution Kabale University
issn 2162-3619
language English
publishDate 2025-07-01
publisher BMC
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series Experimental Hematology & Oncology
spelling doaj-art-eab8238d64c94d04a80ab8a67d817b492025-08-20T03:46:00ZengBMCExperimental Hematology & Oncology2162-36192025-07-011411510.1186/s40164-025-00686-9iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemiaRunze Cai0Binyan Lu1Xiangyu Zhao2Shixin Zhou3Yang Li4Department of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center, Peking UniversityGuangzhou Regenverse Therapeutics Co.,LtdPeking University Institute of Hematology, Peking University People’s HospitalDepartment of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center, Peking UniversityDepartment of Cell Biology, School of Basic Medical Sciences, Peking University Stem Cell Research Center, Peking UniversityAbstract CD226 plays a vital role in NK cell cytotoxicity, interacting with its ligands on tumor targets. Acute myeloid leukemia (AML) cells have developed mechanisms to escape NK cell cytotoxicity, including inducing downregulation of CD226 on NK cells. Induced pluripotent stem cell -derived NK (iPSC-NK) cells offer an important source of standardized off-the-shelf NK cell therapy to treat AML patients. In this study, we engineered iPSC-NK cells with CD226 to assess the ability of killing AML cells. iPSC-NK cells engineered with CD226 have a typical NK cell phenotype and demonstrate improved anti-AML activity and multiple cytokines releasing at low effector-to-target ratios. Transcriptomic analysis revealed upregulation of immune effector function pathways associated with cytotoxicity and immune activation in CD226-overexpression iPSC-NK cells. In an AML xenograft model, mice treated with CD226 overexpression iPSC-NK cells exhibited significantly reduced leukemia burden, prolonged survival, decreased systemic inflammation compared to those treated with Control iPSC-NK cells. Overall, our study provided evidence that iPSC derived-NK cells engineered with CD226 represent a promising candidate for off-the-shelf immunotherapy, particularly in AML and other CD226 ligand-expressing malignancies.https://doi.org/10.1186/s40164-025-00686-9Acute myeloid leukemiaiPSC-derived NK cellsCD226Immunotherapy
spellingShingle Runze Cai
Binyan Lu
Xiangyu Zhao
Shixin Zhou
Yang Li
iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemia
Experimental Hematology & Oncology
Acute myeloid leukemia
iPSC-derived NK cells
CD226
Immunotherapy
title iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemia
title_full iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemia
title_fullStr iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemia
title_full_unstemmed iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemia
title_short iPSC-derived NK cells engineered with CD226 effectively control acute myeloid leukemia
title_sort ipsc derived nk cells engineered with cd226 effectively control acute myeloid leukemia
topic Acute myeloid leukemia
iPSC-derived NK cells
CD226
Immunotherapy
url https://doi.org/10.1186/s40164-025-00686-9
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AT xiangyuzhao ipscderivednkcellsengineeredwithcd226effectivelycontrolacutemyeloidleukemia
AT shixinzhou ipscderivednkcellsengineeredwithcd226effectivelycontrolacutemyeloidleukemia
AT yangli ipscderivednkcellsengineeredwithcd226effectivelycontrolacutemyeloidleukemia