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B7H3-targeting CAR-T cells are an investigational class of cell-based immunotherapies designed to treat a variety of solid tumors and central nervous system (CNS) malignancies. These therapies involve the genetic modification of a patient's T cells to express a chimeric antigen receptor (CAR) that specifically targets B7-H3 (also known as CD276), a transmembrane glycoprotein in the B7 family. B7-H3 is highly overexpressed in numerous cancers—including glioblastoma, neuroblastoma, and various pediatric sarcomas—while maintaining limited expression in normal tissues, making it a promising therapeutic target. Clinical development, notably led by institutions such as Stanford University and Seattle Children's Hospital, has explored regional delivery methods like intracerebroventricular or intracranial infusion to treat brain tumors such as diffuse intrinsic pontine glioma (DIPG), aiming to bypass the blood-brain barrier and minimize systemic toxicity.
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