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AEG35156 is a second-generation synthetic antisense oligonucleotide designed to selectively inhibit the expression of X-linked inhibitor of apoptosis protein (XIAP), a key regulator that prevents apoptosis and is overexpressed in many tumors. By reducing XIAP levels, AEG35156 sensitizes tumor cells to apoptotic signals, including those induced by chemotherapy, thereby potentially overcoming resistance mechanisms in cancer cells. Preclinical and clinical studies have shown that AEG35156 can decrease XIAP mRNA and protein levels both in vitro and in vivo, enhance the efficacy of chemotherapeutic agents such as taxanes or cytarabine/idarubicin, and induce antitumor activity across various cancer models. The drug was primarily investigated for use in acute myeloid leukemia (AML) and other cancers but development has been discontinued.
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