Synucleozid (NSC 377363) 是可编码 α-突触核蛋白的 SNCA mRNA 的有效抑制剂。Synucleozid 选择性靶向 α-synuclein mRNA 的 5'UTR的 IRE 位点，减少了加载到多核糖体中的 SNCA mRNA 的量，从而抑制了α-synuclein 蛋白的翻译。Synucleozid 有潜力用于帕金森病的相关研究。

Synucleozid hydrochloride (NSC 377363 hydrochloride) is a potent inhibitor of the SNCA mRNA that encodes α-synuclein protein. Synucleozid selectively targets the α-synuclein mRNA 5′ UTR at the designed IRE site, decreases the amount of SNCA mRNA loaded into polysomes and thereby inhibits SNCA translation. Synucleozid has the potential for the investigation of Parkinson’s disease.

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Synucleozid (0.25-1 μM; 24 hours) abrogates cytotoxicity induced by α-synuclein preformed fibrils, which act as seeds and recruit endogenous α-synuclein to aggregate.
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 with an IC50 of 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 with an EC50 value of 2.7 ± 0.4 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 ± 0.3 μM.
Synucleozid (0.25-1 μM; 24 hours) decreases α-synuclein and other proteins that have IREs in the

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4°C, sealed storage, away from moistur In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

[1]. Zhang P, et al. Translation of the intrinsically disordered protein α-synuclein is inhibited by a small molecule targeting its structured mRNA.Proc Natl Acad Sci U S A. 2020 Jan 21;117(3):1457-1467.

In Vitro: DMSO : 125 mg/mL (308.72 mM; Need ultrasonic)H₂O : 2 mg/mL (4.94 mM; Need ultrasonic)配制储备液