NS13001 是一种有效的、选择性的、具有口服活性 SK channels (小电导钙激活钾通道) 正向变构调节剂。NS13001 对 SK2 和 SK3 通道的 EC50 分别为 1.8 μM 和 0.14 μM。NS13001 有望作为治疗脊髓小脑共济失调 2 型 (SCA2) 和可能的其他小脑共济失调的潜在治疗剂。
NS13001 is a potent, selective, orally active allosteric positive modulator of SK channels (small conductance calcium-activated potassium channels). The EC50s are 1.8 and 0.14 μM for SK2 and SK3, respectively. NS13001 holds promise as a potential therapeutic agent for treatment of spinocerebellar ataxia type 2 (SCA2) and possibly other cerebellar ataxias[1].
[1]. Kasumu AW, et al. Selective positive modulator of calcium-activated potassium channels exerts beneficial effects in a mouse model of spinocerebellar ataxia type 2. Chem Biol. 2012 Oct 26;19(10):1340-53.
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