A novel way to reduce ASO toxicity
mPCS1 is a fully PS-modified 14-mer LNA gapmer antisense oligonucleotide (ASO) with two phosphodiester-linked GalNAc units. mPCS1 targets mouse Pcsk9 (Proprotein To assess the molecular impact of BS on ASO toxicity, RNA sequencing (RNA-Seq) analysis was performed. Specifically, C57BL/6J mice were subcutaneously treated with 100 nmol/kg of mPCS1 or BRO and liver global gene expression was assessed 72 h after administration Day 3 was selected for RNA-seq analysis.