计算溶液所需的质量、体积或浓度。
为什么选择此级别
，适用于对基线干扰要求严格的色谱和分析工作流程。
储存与运输
-20°C储存。超低温运输 。请查阅批次 COA 获取详细规格。
质量文档
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文献证明
在色谱分析、有机合成和交叉偶联反应领域已被 0 篇同行评审文献引用。
概述
Rugonersen (RG6091; RO7248824) is a locked-nucleic acid (LNA)- modified antisense oligonucleotides (ASOs), and results in reduction of ubiquitin-protein ligase E3A (UBE3A) silencing. Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A, Rugonersen has been used for AS reasearch
In Vitro
RO7248824 (0-10 μM) show a nanomolar potency against UBE3A-ATS (EC 50 =26.3 nM), UBE3A mRNA upregulation (EC 50 =15.4 nM) and UBE3A protein upregulation (EC 50 =24.8 nM) in Angelman syndrome (AS) neurons. MCE has not independently confirmed the accuracy of these methods. They are for reference only.
In Vivo
Rugonersen (RO7248824) (24 mg/monkey; i.t.; for 8-85 d) is well tolerated without adverse in-life effects or tissue pathology and produced a robust, long lasting (up to 3 months) paternal reactivation of UBE3A mRNA/protein across key monkey brain regions . Male cynomolgus monkeys Rugonersen (150 μg; i.c.v.; single dose) selectively and potently reduces UBE3A-ATS, while concomitantly upregulating the UBE3A mRNA and protein . MCE has not independently confirmed the accuracy of these methods. They are for reference only. Animal Model: Male cynomolgus monkey Dosage: 24 mg per monkey Administration: Intrathecal injection; single dose or twice dose with 2 weeks apart; sacrificed at 8, 15, 29, 57, and 85 days after the last dose Result: Resulted a long duration of action on paternal UBE3A reactivation in NHP brains after IT delivery. Animal Model: WT and AS Ube3a m- / p+ mice adult mice (10-12 weeks old) Dosage: 150 μg per mice Administration: Intracerebroventricular injection; single dose; harvested at 2 weeks post injection Result: Revealed a steep relationship between UBE3A-ATS knock-down and UBE3A mRNA/protein upregulation, whereby an almost 90% downregulation was needed to achieve a 50% upregulation, respectively.
Form:Solid
IC50& Target:ubiquitin-protein ligase E3A (UBE3A)
规格
别名
鲁格纳森
英文名称
Rugonersen
生化机理
Rugonersen（RG6091；RO7248824）是一种经锁定核酸（LNA）修饰的反义寡核苷酸（ASO），可减少泛素蛋白连接酶 E3A（UBE3A）的沉默。安杰曼综合征（Angelman syndrome，AS）是一种严重的神经发育障碍性疾病，由以下原因引起
储存条件
-20°C储存
运输条件
超低温运输
名称和识别符
分子类型
小分子
Smiles
[Rugonersen]
分子量
6530.30
技术文档
高级数据
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溶液计算器
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