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UGT1A1 antisense morpholino oligonucleotide is a synthetic antisense agent belonging to the phosphorodiamidate morpholino oligonucleotide (PMO) class. It is designed to specifically target and inhibit the translation of the UDP-glucuronosyltransferase 1-1 (UGT1A1) enzyme, which is responsible for the glucuronidation and subsequent excretion of bilirubin. By reducing UGT1A1 activity, the drug induces a controlled state of physiological unconjugated hyperbilirubinemia. This elevation in bilirubin levels provides therapeutic benefits due to bilirubin's role as a potent endogenous antioxidant and anti-inflammatory signaling molecule. In preclinical models of cyclosporine A (CsA)-induced chronic kidney disease, the administration of this morpholino has been shown to protect renal function, activate the nuclear receptor PPAR-α, and downregulate pro-inflammatory and injury markers such as NF-κB, Kim-1, and NGAL.
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