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SGT-53 is a tumor-targeted gene therapy nanomedicine designed to deliver a plasmid encoding wild-type human p53 directly to tumor cells. The drug uses cationic liposomes decorated with a transferrin receptor-targeting single-chain variable fragment (scFv) antibody for selective delivery across the blood-brain barrier and into tumors that overexpress the transferrin receptor. By restoring functional p53 expression in cancer cells—where this tumor suppressor is often mutated or inactivated—SGT-53 stimulates apoptosis and immunogenic cell death, enhances anti-tumor immunity, and can sensitize tumors to chemotherapy and immunotherapy (such as anti-PD1 agents). It has been studied in various solid tumors including glioblastoma, pancreatic cancer, breast cancer, medulloblastoma, choroid plexus carcinoma, and other CNS malignancies. Developed by SynerGene Therapeutics[3][4][5][6].
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