Tofersen (BIIB067) 是一种反义寡核苷酸,可介导超氧化物歧化酶 1 () 的 RNase H 依赖性降解,以减少 SOD1 蛋白的合成。Tofersen 可用于肌萎缩侧索硬化 (ALS) 的研究。 DNA, d([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2- methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]rA-sp-Tsp-A-sp-m5C-sp-A-sp-T-sp-T-sp-T-sp-m5C-sp-T-sp-A-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU)