Dnm2 downregulation by antisense oligonucleotide (ASO1) injections rescue a murine model of recessive centronuclear myopathy (Bin1mck-/-)

In this study, we performed RNA sequencing on tibialis anterior muscle of male mice. WT (injected with PBS) and Bin1mck-/- (injected with PBS) mice as well as ASO1 injected Bin1mck-/- mice and ASO1 injected WT mice were sequenced at 7 weeks of age. ASO1 targets specifically Dnm2 ARN. The study is composed of 4 groups of mice: WT PBS, Bin1mck-/- PBS, WT ASO1 and Bin1mck-/- ASO1. Each group was composed of quadruplicates. The comparison of transcriptome profiles across the different genotypes was assessed in this study.

本研究针对雄性小鼠的胫前肌（tibialis anterior muscle）开展了RNA测序（RNA sequencing）。注射磷酸盐缓冲液（PBS）的野生型（WT）小鼠、注射PBS的Bin1mck-/-纯合敲除小鼠，以及经ASO1处理的Bin1mck-/-纯合敲除小鼠与经ASO1处理的WT小鼠，均于7周龄时接受测序。ASO1可特异性靶向Dnm2的RNA（原文疑似笔误，将RNA写为ARN）。本研究共设置4组小鼠：WT PBS组、Bin1mck-/- PBS组、WT ASO1组及Bin1mck-/- ASO1组，每组均包含4个生物学重复。本研究旨在比较不同基因型小鼠的转录组谱（transcriptome profiles）。