Synucleozid-2.0 is a blood-brain barrier-permeable inhibitor that binds to the IRE of SNCA mRNA , with a EC 50 of 2.9 µM and a K d value of 1.8 µM. Synucleozid-2.0 selectively binds to and stabilizes the A bulge and adjacent closed base pairs in the 5' UTR IRE of SNCA mRNA , blocks the translation process and reduces intracellular levels of α-synuclein . Synucleozid-2.0 exerts cytoprotective effects against cytotoxicity induced by α-synuclein preformed fibrils. Synucleozid-2.0 is applicable to the research of Parkinson's disease.
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