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ELX-02 disulfate (Synonyms:NB-124 disulfate)
目录号 : KM4569 CAS No. : 2244622-33-9 纯度 : ≥98%

ELX-02 disulfate (NB-124 disulfate) 是一种首创的的合成的真核核糖体特异性糖苷 (ERSG),可用于研究无义突变引起的遗传疾病。

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5mg
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10mg
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50mg 询价 In-stock
100mg 询价 In-stock
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生物活性

ELX-02 disulfate (NB-124 disulfate) is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). ELX-02 disulfate is being developed as a therapy for genetic diseases caused by nonsense mutations.

体外研究

ELX-02 (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells.

Cell Cytotoxicity Assay

Cell Line: Wildtype human proximal tubule cells (HK-2)
Concentration: 0, 100 and 400 μg/mL
Incubation Time: 0, 24, 48 and 72 hours
Result: Cytotoxicity assay in wildtype human proximal tubule cells (HK-2) showing no toxic effect of 400 μg/mL at 0, 24, 48 and 72 h.
体内研究

ELX-02 (10 and 30 mg/kg; repeat subcutaneous administration; twice weekly, total of 8 doses) shows accumulation in tissues that is dose dependent without gender difference.
In plasma ELX-02 is rapidly absorbed with a Tmax of 0.25 h after both single (a single subcutaneous injection at 10 mg/kg at dose volume of 5 mL/kg) and repeated administration (twice weekly with 10 mg/kg/dose for 21 days; total of 7 administrations). ELX-02 is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h.
In a Ctns nonsense mutant mouse, subcutaneous ELX-02 accumulates in kidney tissue without overt renal toxicity and that ELX-02 (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo.

Animal Model: Twenty-nine Ctns mice 5-7 month old
Dosage: 10 and 30 mg/kg
Administration: Subcutaneous injection, at dose volume of 5 mL/kg, twice weekly for a period of 28 days (total of 8 doses)
Result: Highest levels were measured in the kidney, followed by spleen and liver, with lower levels in other tissues (lung, heart, cochlea and brain).
分子式
C19H40N4O14S
分子量
678.68
CAS号
2244622-33-9
运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

-20°C, stored under nitrogen

*In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)

溶解性数据
In Vitro: 

H2O : 100 mg/mL (147.34 mM; Need ultrasonic)

DMSO : < 1 mg/mL (insoluble or slightly soluble)

配制储备液
浓度 溶剂体积 质量 1 mg 5 mg 10 mg
1 mM 1.4734 mL 7.3672 mL 14.7345 mL
5 mM 0.2947 mL 1.4734 mL 2.9469 mL
10 mM 0.1473 mL 0.7367 mL 1.4734 mL
*

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 (stored under nitrogen)。-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。

In Vivo:

请根据您的实验动物和给药方式选择适当的溶解方案。以下溶解方案都请先按照 In Vitro 方式配制澄清的储备液,再依次添加助溶剂:

——为保证实验结果的可靠性,澄清的储备液可以根据储存条件,适当保存;体内实验的工作液,建议您现用现配,当天使用; 以下溶剂前显示的百
分比是指该溶剂在您配制终溶液中的体积占比;如在配制过程中出现沉淀、析出现象,可以通过加热和/或超声的方式助溶

  • 1.

    请依序添加每种溶剂: PBS

    Solubility: 100 mg/mL (147.34 mM); Clear solution; Need ultrasonic

临床试验
The molarity calculator equation
Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)
The dilution calculator equation
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
This equation is commonly abbreviated as: C1V1 = C2V2
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