Tominersen (RG6042) 是第二代 2′-O-(2-甲氧基乙基) 反义寡核苷酸，靶向亨廷蛋白 (HTT) mRNA，有效抑制 HTT 的合成。Tominersen 可用于亨廷顿病的研究。
货号:ajcx38460
CAS:1709886-74-7
分子式:N/A
分子量：7078
溶解度:N/A
纯度：98%
存储：Store at -20°C
库存： 现货

Background:

Tominersen (RG6042) is a second-generation 2′-O-(2-methoxyethyl) antisense oligonucleotide that targets huntingtin protein (HTT) mRNA and potently suppresses HTT production. Tominersen can be used for the research of Huntington's disease[1].

Tominersen binds to its cognate mRNA by means of Watson-Crick base-pair interactions, triggering RNase H1-mediated degradation of the target mRNA[1].

[1]. Tabrizi SJ, et, al. Targeting Huntingtin Expression in Patients with Huntington's Disease. N Engl J Med. 2019 Jun 13;380(24):2307-2316.