Synucleozid hydrochloride is a potent the SNCA mRNA inhibitor that encodes α-synuclein protein (IC50=1.5 μM), has the potential for the investigation of Parkinson’s disease.

SNCA IRE RNA:1.5 μM

Synucleozid (0.25-1 μM; 24 hours) eliminates α-synuclein preformed fibrils induced cytotoxicity, fibrils act as seeds and recruit endogenous α-synuclein aggregation.Synucleozid (0-1 μM; 24 hours) binds to the A bulge near the base of the IRE hairpin, reduced levels of α-synuclein in a dose-dependent manner(IC50 : 500 nM), and inhibits α-synuclein protein expression in SH-SY5Y neuroblastoma cells.Synucleozid (100 nM-100 μM; 24 hours) binds to 2-AP-labeled and native IRE RNA with similar affinities. It decreases 2-AP emission(EC50?value of 2.7 μM), recovery of 2-AP emissions is observed as a function of unlabeled?SNCA?IRE RNA (RNA-0) concentration, affording a competitive?Kd?of 1.5 μM.Synucleozid (0.25-1 μM; 24 hours) decreases α-synuclein and other proteins that have IREs in their mRNA's UTR including APP, PrP, Ferritin and TfR as a dose-dependent mannner. All panels is completed in SH-SY5Y cells, except for PrP protein which is assessed in Neuro-2A cells.