GPC2 has been suggested as a therapeutic target in neuroblastoma. GPC2 is highly expressed in about half of neuroblastoma cases and that high GPC2 expression correlates with poor overall survival. GPC2 silencing inactivates Wnt/β-catenin signaling and reduces the expression of N-Myc, an oncogenic driver of neuroblastoma tumorigenesis. Immunotoxins and chimeric antigen receptor (CAR) T cells targeting GPC2 inhibit neuroblastoma growth in mouse models. A GPC3 specific antibody drug conjugate (ADC) can also inhibit neuroblastoma cell proliferation.
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