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LV-shCD81 is a lentiviral vector-based gene therapy designed to suppress the expression of the tetraspanin protein CD81 through RNA interference (RNAi). CD81 is a cell surface protein that is frequently overexpressed in various hematological malignancies, including non-Down syndrome acute megakaryoblastic leukemia (AMKL), where it contributes to tumor cell proliferation, survival, and metastasis. The vector delivers a specific short hairpin RNA (shRNA) sequence that targets CD81 mRNA for degradation, thereby reducing the levels of CD81 protein on the cell surface. Preclinical research indicates that knockdown of CD81 using this lentiviral approach inhibits AMKL cell proliferation, induces apoptosis, and alters cell cycle progression by decreasing the proportion of cells in the S phase. This agent is being explored as a potential targeted therapy for AMKL and other CD81-overexpressing cancers.
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