2. Metabolic Enzyme/Protease
  3. E1/E2/E3 Enzyme
  4. Rugonersen

Rugonersen  (Synonyms: RG6091; RO7248824)

目录号: HY-148130
产品使用指南

摩尔计算器

Rugonersen (RG6091; RO7248824) 是一种锁定核酸 (LNA) 修饰的反义寡核苷酸 (ASOs)，可减少泛素蛋白连接酶 E3A (UBE3A) 沉默。Angelman 综合征 (AS) 是一种由神经元 E3连接酶 UBE3A 缺失引起的严重神经发育障碍，Rugonersen 已用于 AS 的研究。

该游离形式化合物不稳定，推荐具有相同生物学活性的稳定盐形式 Rugonersen sodium。

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DNA, d(P-thio)((2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)rA-C-(2′-O,4′-C-methylene)rA-C-T-T-A-A-T-T-A-T-A-C-T-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)m5rC)

Rugonersen Chemical Structure

CAS No. : 2591587-57-2

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规格 价格 是否有货
1 mg ￥1140
询问价格 & 货期
5 mg ￥3170
询问价格 & 货期
10 mg ￥5080
询问价格 & 货期

* Please select Quantity before adding items.

Rugonersen 的其他形式现货产品:

Customer Review

Other Forms of Rugonersen:

Rugonersen 相关抗体

Top Publications Citing Use of Products

  • 生物活性

  • 纯度 & 产品资料

  • 参考文献

生物活性

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[1][2].

IC50 & Target

ubiquitin-protein ligase E3A (UBE3A)[1]
体外研究
(In Vitro)

RO7248824 (0-10 μM) show a nanomolar potency against UBE3A-ATS (EC50=26.3 nM), UBE3A mRNA upregulation (EC50=15.4 nM) and UBE3A protein upregulation (EC50=24.8 nM) in Angelman syndrome (AS) neurons[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Rugonersen 相关抗体:
体内研究
(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[1].
Male cynomolgus monkeys[1] Rugonersen (150 μg; i.c.v.; single dose) selectively and potently reduces UBE3A-ATS, while concomitantly upregulating the UBE3A mRNA and protein[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: Male cynomolgus monkey[1]
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)[1]
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.
Clinical Trial
分子量

6530.30

CAS 号
性状

固体

颜色

White to off-white

Sequence

DNA, d(P-thio)((2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)rA-C-(2′-O,4′-C-methylene)rA-C-T-T-A-A-T-T-A-T-A-C-T-(2′-O,4′-C-methylene)m5rU-(2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)m5rC)
运输条件

Room temperature in continental US; may vary elsewhere.
储存方式

-20°C, sealed storage, away from moisture

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

溶解性数据
细胞实验： 

H2O 中的溶解度 : ≥ 100 mg/mL (15.31 mM)

* "≥" means soluble, but saturation unknown.

配制储备液
浓度 溶剂体积 质量 1 mg 5 mg 10 mg
1 mM 0.1531 mL 0.7657 mL 1.5313 mL
5 mM 0.0306 mL 0.1531 mL 0.3063 mL
查看完整储备液配制表

* 请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液；一旦配成溶液，请分装保存，避免反复冻融造成的产品失效。
储备液的保存方式和期限：-80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)。-80°C储存时，请在6个月内使用，-20°C储存时，请在1个月内使用。

* 备注：如您选择水作为储备液，请稀释至工作液后，再用 0.22 μm 的滤膜过滤除菌后使用。

  • 摩尔计算器

  • 稀释计算器

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

质量
=
浓度
×
体积
×
分子量 *

Concentration (start) × Volume (start) = Concentration (final) × Volume (final)

This equation is commonly abbreviated as: C1V1 = C2V2

浓度 (start)

C1

×
体积 (start)

V1

=
浓度 (final)

C2

×
体积 (final)

V2

动物溶解方案计算器
请输入动物实验的基本信息：

给药剂量

mg/kg

动物的平均体重

g

每只动物的给药体积

μL

动物数量

只
由于实验过程有损耗，建议您多配一只动物的量
计算结果
工作液所需浓度 : mg/mL
该产品水溶性佳，请具体参考实测 水 / PBS / Saline 中的溶解度数据。
您所需的储备液浓度超过该产品的实测溶解度，如有需要，请与 MCE 中国技术支持联系。
纯度 & 产品资料
参考文献

Rugonersen 相关分类

完整储备液配制表

* 请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液；一旦配成溶液，请分装保存，避免反复冻融造成的产品失效。
储备液的保存方式和期限：-80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)。-80°C储存时，请在6个月内使用，-20°C储存时，请在1个月内使用。

可选溶剂 浓度 溶剂体积 质量 1 mg 5 mg 10 mg 25 mg
H2O 1 mM 0.1531 mL 0.7657 mL 1.5313 mL 3.8283 mL
5 mM 0.0306 mL 0.1531 mL 0.3063 mL 0.7657 mL
10 mM 0.0153 mL 0.0766 mL 0.1531 mL 0.3828 mL
15 mM 0.0102 mL 0.0510 mL 0.1021 mL 0.2552 mL

* 备注：如您选择水作为储备液，请稀释至工作液后，再用 0.22 μm 的滤膜过滤除菌后使用。

Help & FAQs
  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Rugonersen2591587-57-2RG6091 RO7248824RG 6091RG-6091RO7248824RO 7248824RO-7248824E1/E2/E3 EnzymeE1 activating enzymeE2 conjugating enzymeE3 ligating enzymeUbiquitin activating enzymeUbiquitin conjugating enzymeUbiquitin ligaseAngelman syndromeUBE3A-ATSlong lasting effectcynomolgus monkey AS human pluripotent stem cell neuronsInhibitorinhibitorinhibit

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