Rovanersen (WVE-120101) 是一种反义寡核苷酸，通过特异靶向 huntingtin (HTT) 基因的突变mRNA拷贝，而不影响正常的 HTT基因的mRNA，从而防止有缺陷的 huntingtin 蛋白的产生。Rovanersen 可用于亨廷顿病的研究。
货号:ajcx38438
CAS:2072901-32-5
分子式:N/A
分子量：6617
溶解度:N/A
纯度：98%
存储：Store at -20°C
库存：现货

Background:

Rovanersen (WVE-120101) is an antisense oligonucleotide that specifically targets mutated mRNA copies of the huntington (HTT) gene without affecting healthy mRNA of HTT gene, thereby preventing the production of faulty Huntingtin protein. Rovanersen can be used for huntington's disease research[1].

In vitro, Rovanersen (WVE-120101) leads to the selective RNase H cleavage of mutant Huntingtin allele (mHTT) over wtHTT, with no complement activation. Rovanersen selectively decreases mHTT mRNA and protein levels compared with wtHTT in multiple cells lines[1].

In non-human primates (NHPs), Rovanersen (WVE-120101) is found in nuclear and perinuclear compartments of neurons in brain areas believed to be involved in HD pathology[1].

[1]. M Meena, et al. Selectivity and Biodistribution of WVE-120101, a Potential Antisense Oligonucleotide Therapy for the Treatment of Huntington's Disease (S56.001). AAN Enterprises. April 18, 2017; 88 (16 Supplement).