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**GPC2-targeted CAR-T cell therapy** is an investigational cell therapy where patient or donor T cells are genetically engineered to express a chimeric antigen receptor (CAR) specific for the tumor-associated antigen glypican-2 (GPC2), a cell-surface protein highly expressed in pediatric neuroblastoma and certain central nervous system embryonal tumors[2][8]. The CAR-T cells recognize and bind to GPC2 on tumor cells, leading to immune-mediated cytotoxicity. Preclinical engineering (notably using the CT3 single-chain variable fragment, CD28 hinge/transmembrane, and 4-1BB co-stimulatory domains) has maximized anti-tumor efficacy in neuroblastoma models[2]. Clinical trials are evaluating safety, dosing, and preliminary efficacy in relapsed/refractory neuroblastoma and medulloblastoma[1][3][4][7][9].
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