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GQC-05 is a small molecule G-quadruplex interacting drug (GQID) that functions by stabilizing DNA secondary structures known as G-quadruplexes. These structures are located in the promoter regions of key oncogenes, specifically *MYC* (c-Myc) and *BCL2*. By stabilizing these G-quadruplexes, GQC-05 effectively inhibits the transcription of these genes, leading to a significant reduction in MYC and Bcl-2 mRNA and protein levels. This downregulation induces apoptosis and inhibits cell growth in various cancer models, particularly acute myeloid leukemia (AML). Research indicates that GQC-05 may also act as a DNA damaging agent and shows synergistic activity when combined with Bcl-2 inhibitors such as navitoclax.
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