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Small interfering RNA targeting TBCE is an experimental nucleic acid therapeutic designed to silence the expression of tubulin folding cofactor E (TBCE). TBCE is a chaperone protein essential for the folding of alpha-tubulin and the subsequent formation of alpha/beta-tubulin heterodimers, which are the building blocks of microtubules. Research indicates that elevated TBCE expression is a significant driver of resistance to platinum-based chemotherapies, such as cisplatin, in hepatocellular carcinoma (HCC). By utilizing RNA interference (RNAi) to knock down TBCE mRNA, this therapeutic disrupts cytoskeleton rearrangement and microtubule dynamics, thereby sensitizing cancer cells to DNA-damaging agents and promoting apoptosis. In preclinical studies, it has been delivered via endosomal pH-responsive nanoparticles in combination with cisplatin prodrugs to overcome chemoresistance in orthotopic and patient-derived xenograft (PDX) models of liver cancer.
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