Rovanersen (Synonyms: WVE-120101) |
| カタログ番号GC64860 |
Rovanersen (WVE-120101) は、ハンチントン (HTT) 遺伝子の正常な mRNA に影響を与えることなく、ハンチントン (HTT) 遺伝子の変異した mRNA コピーを特異的に標的とするアンチセンス オリゴヌクレオチドです。
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Cas No.: 2072901-32-5
Sample solution is provided at 25 µL, 10mM.
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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).
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