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CART-EGFRvIII is an autologous chimeric antigen receptor (CAR) T cell therapy engineered to target the epidermal growth factor receptor variant III (EGFRvIII), a tumor-specific mutation commonly found in glioblastoma multiforme (GBM). Patient-derived T cells are genetically modified ex vivo using lentiviral transduction to express a CAR specific for EGFRvIII. These modified T cells are expanded and reinfused into the patient, where they recognize and kill EGFRvIII-expressing tumor cells. The therapy has demonstrated feasibility and safety in early-phase clinical trials for recurrent glioblastoma, with evidence of on-target activity and minimal off-tumor toxicity. Mechanistically, CART-EGFRvIII mediates direct cytotoxicity against tumor cells expressing the mutant receptor and may induce antigen loss variants as part of its immunological effect[2][3][8].
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