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Exaluren sulfate  (Synonyms: ELX-02 sulfate; NB-124 sulfate)

目录号: HY-114231C
产品使用指南

Exaluren (ELX-02) sulfate 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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Exaluren sulfate Chemical Structure

Exaluren sulfate Chemical Structure

CAS No. : 1375073-94-1

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MCE 顾客使用本产品发表的 1 篇科研文献

  • 生物活性

  • 纯度 & 产品资料

  • 参考文献

生物活性

Exaluren (ELX-02) sulfate is an investigational, advanced synthetic eukaryotic ribosome selective glycoside (ERSG). Exaluren sulfate is being developed as a therapy for genetic diseases caused by nonsense mutations[1].

体外研究
(In Vitro)

Exaluren (ELX-02) sulfate (100-400 μg/mL) is not toxic, and permits read-through of nonsense mutations in human cells[1].

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

Cell Cytotoxicity Assay[1]

Cell Line: Wildtype human proximal tubule cells (HK-2)
Concentration: 0, 100 and 400 μg/mL
Incubation Time: 0, 24, 48 and 72 h
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
(In Vivo)

Exaluren (ELX-02) sulfate (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[1].
In plasma Exaluren (ELX-02) sulfate 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). Exaluren (ELX-02) sulfate is rapidly eliminated from plasma in a biphasic manner with the terminal half-life (T1/2) of 0.5 h[1].
In a CtnsY226X nonsense mutant mouse, subcutaneous Exaluren (ELX-02) sulfate accumulates in kidney tissue without overt renal toxicity and that Exaluren (ELX-02) sulfate (10 mg/kg X2/week for 3 weeks) reduces renal cystine accumulation in vivo[1].

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

Animal Model: Twenty-nine CtnsY226X/Y226X mice 5-7 month old[1]
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).
Clinical Trial
分子量

580.60

Formula

C19H40N4O14S

CAS 号
运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

Please store the product under the recommended conditions in the Certificate of Analysis.

纯度 & 产品资料
参考文献

Exaluren sulfate 相关分类

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  • 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.

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Exaluren sulfate
目录号:
HY-114231C
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