您好,欢迎来到试剂仪器网! [登录] [免费注册]
试剂仪器网
位置:首页 > 产品库 > Tofersen
立即咨询
咨询类型:
     
*姓名:
*电话:
*单位:
Email:
*留言内容:
请详细说明您的需求。
*验证码:
 
Tofersen
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。
CAS NO:2088232-70-4
包装与价格:
包装价格(元)
5mg电议
10mg电议

产品介绍
Tofersen (BIIB067) 是一种反义寡核苷酸,可介导超氧化物歧化酶 1 (SOD1) mRNA 的 RNase H 依赖性降解,以减少 SOD1 蛋白的合成。Tofersen 可用于肌萎缩侧索硬化 (ALS) 的研究。
Cas No.2088232-70-4
别名BIIB067; ISIS-SOD1Rx
分子量7128
溶解度Water : 100 mg/mL (14.03 mM; Need ultrasonic)
储存条件-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

Tofersen (BIIB067) is an antisense oligonucleotide that mediates RNase H-dependent degradation of superoxide dismutase 1 (SOD1) mRNA to reduce the synthesis of SOD1 protein. Tofersen can be used for the research of amyotrophic lateral sclerosis (ALS)[1].

Tofersen targets the SOD1 mRNA using antisense oligonucleotides (ASOs) that bind to the SOD1 mRNA by Watson-Crick base pairing. Tofersen activates RNase H1 that destroys the targeted RNA[2].

[1]. Miller T, et, al. Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS. N Engl J Med. 2020 Jul 9;383(2):109-119. [2]. McCampbell A, et, al. Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models. J Clin Invest. 2018 Aug 1;128(8):3558-3567.