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GO-201 is a synthetic peptide inhibitor that directly targets the Mucin 1 (MUC1) oncoprotein, which is aberrantly overexpressed in approximately 90% of human breast cancers. GO-201 binds to the MUC1 cytoplasmic domain, blocking the formation of MUC1 oligomers, attenuating nuclear localization, disrupting redox balance, and activating DNA damage response in cancer cells. This leads to growth arrest and necrotic cell death in MUC1-expressing tumor cells, with no effect on nonmalignant mammary or MUC1-null cells. In preclinical models, GO-201 showed efficacy in inducing tumor regression.
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