Tofersen的临床研究进展

Clinical research advances of Tofersen

Tofersen是一种鞘内给药的反义寡核苷酸药物,用于治疗肌萎缩侧索硬化症(ALS),其旨在通过诱导RNase H介导的超氧化物歧化酶1(SOD1)mRNA降解来减少SOD1蛋白的合成。本文着重对首款针对ALS的基因靶向疗法Tofersen的临床研究进展进行论述。

Tofersen is an antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis(ALS).Tofersen causes degradation of SOD1 mRNA through binding to SOD1 mRNA,which results in a reduction of SOD1 protein synthesis.This article focuses on an overview of the clinical research advances of Tofersen,the first gene-targeted therapy for ALS.