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CAR123 T lymphocytes are autologous or allogeneic T cells genetically engineered to express a chimeric antigen receptor (CAR) that specifically targets CD123, the interleukin-3 receptor alpha chain. CD123 is highly expressed on the surface of malignant hematopoietic cells in acute myeloid leukemia (AML) and other myeloid malignancies. Upon infusion into patients, these modified T lymphocytes recognize and bind to CD123-expressing tumor cells, leading to their activation and targeted cytotoxicity against cancerous cells. The mechanism involves direct cell-mediated killing via recognition of the target antigen by the engineered receptor. This therapy is under investigation for its potential in treating relapsed or refractory AML and other hematologic cancers expressing CD123[1][2][3][4]. Various strategies are being explored to improve safety and efficacy, including modular/switchable platforms (e.g., UniCAR), dual-targeting approaches, non-viral manufacturing methods (e.g., piggyBac transposon), and logic-gated/inhibitory designs to reduce off-tumor toxicity[1][3][6].
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