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25CAR T-cells are an experimental chimeric antigen receptor (CAR) T-cell therapy targeting CD25 (the alpha chain of the IL-2 receptor), primarily developed for the treatment of poor-prognosis acute myeloid leukemia (AML). Research conducted by RIKEN and collaborating institutions identified CD25 as a marker over-represented in AML-initiating cells compared to normal hematopoietic stem and progenitor cells. To address the challenge of leukemic cells residing in the bone marrow, a modified version known as CXCR4-25CAR was engineered to co-express the chemokine receptor CXCR4, enhancing the homing and targeting of AML cells within the bone marrow niche. Preclinical studies in patient-derived xenograft (PDX) models demonstrated that these CAR T-cells could effectively eliminate AML cells in both peripheral blood and bone marrow without significant off-target toxicity or xenogeneic graft-versus-host disease.
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