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).
In Vitro
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. MCE has not independently confirmed the accuracy of these methods. They are for reference only.
Form:Solid
规格
别名
托弗森
规格或纯度
≥95%
英文名称
Tofersen
生化机理
Tofersen(BIIB067)是一种反义寡核苷酸,可介导 RNase H 依赖性降解超氧化物歧化酶 1(SOD1)mRNA,从而减少 SOD1 蛋白的合成。Tofersen 可用于肌萎缩性脊髓侧索硬化症(ALS)的研究。