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ELX-02 disulfate
本产品不向个人销售,仅用作科学研究,不用于任何人体实验及非科研性质的动物实验。
ELX-02 disulfate图片
CAS NO:2244622-33-9
包装与价格:
包装价格(元)
10mM (in 1mL Water)电议
1mg电议
5mg电议
10mg电议

产品介绍
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。
Cas No.2244622-33-9
别名NB-124 disulfate
分子式C19H42N4O18S2
分子量678.68
溶解度Water : 100 mg/mL (147.34 mM; Need ultrasonic)|DMSO :< 1 mg/mL (insoluble or slightly soluble)
储存条件Store at -20°C, stored under nitrogen
General tipsFor obtaining a higher solubility , please warm the tube at 37 ℃ and shake it in the ultrasonic bath for a while.
Shipping ConditionEvaluation sample solution : ship with blue ice
All other available size: ship with RT , or blue ice upon request
产品描述

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[1].

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

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[1].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[1]. In a CtnsY226X 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[1].

[1]. Leubitz A, et al. Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of ELX-02, a Potential Treatment for Genetic Disorders Caused by Nonsense Mutations, in Healthy Volunteers. Clin Pharmacol Drug Dev. 2019 Jan 16.