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AC-915 is a novel amino-trifluoro-phthalimide analog being investigated for the treatment of hepatocellular carcinoma (HCC). It functions by binding to lipid droplets and the endoplasmic reticulum (ER) membrane, where it interacts with several chaperone proteins, including HSP60, HSP70, HSP90, and protein disulfide isomerase (PDI). AC-915 specifically inhibits PDI activity, leading to the induction of cell death in HCC cells and the inhibition of cell proliferation and migration. In vivo studies in mouse models have demonstrated that the compound preferentially accumulates in the liver and significantly reduces tumor incidence and size. The compound possesses inherent blue fluorescence, which facilitates biodistribution analysis in native tissue sections.
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