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MitoBloCK-6 (MB-6) is a small molecule inhibitor of the mitochondrial disulfide relay system, specifically targeting the FAD-linked sulfhydryl oxidase known as Augmenter of Liver Regeneration (ALR), also referred to as GFER or Erv1. Developed through a collaboration between UCLA and the University of Florence, MitoBloCK-6 disrupts the Mia40/Erv1 redox-mediated protein import pathway into the mitochondrial intermembrane space. This inhibition leads to mitochondrial dysfunction, cytochrome c release, and the induction of apoptosis. Preclinical research has demonstrated its potential as an antineoplastic agent, showing selective cytotoxicity against hepatocellular carcinoma and acute myeloid leukemia cells while sparing differentiated cells. It is primarily used as a research tool to investigate mitochondrial protein import and has been administered via intraperitoneal injection in animal models.
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