Tofersen的临床研究进展
Clinical research advances of Tofersen
王亚茹 1王丽双 1温雅静 1阚红亮 1章晓骅1
作者信息
- 1. 南京正大天晴制药有限公司,南京 210046
- 折叠
摘要
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.
Abstract
Tofersen is an antisense oligonucleotide indicated for the treatment of amyotrophic lateral sclerosis(ALS).Tofersen causes degra-dation 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.
关键词
反义寡核苷酸/肌萎缩侧索硬化症/Tofersen/SOD1蛋白Key words
Antisense oligonucleotides/Amyotrophic lateral sclerosis/Tofersen/SOD1 protein引用本文复制引用
出版年
2024
NETL
NSTL
万方数据