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.
Antisense oligonucleotidesAmyotrophic lateral sclerosisTofersenSOD1 protein
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