Gates Package — page 857

Fig.11. Differentially expressed genes post in vivo base editing HDAd-EF1a.ABE8e. RNA-seq analysis was performed with bone marrow MNCs collected before treatment and at week 16 after in vivo HSC transduction/selection with HDAd-EF1a-ABE8e (N=3 females, age 8-12 weeks). The genome of CD46/YAC mice contains the wild-type 248-kb -globin locus (-YAC) including the LCR, -, d-, and -globin genes. Shown are genes with altered mRNA expression based on their adjusted p-value after annotation with the human genome. A) Altered mRNA expression after annotation with a reference human genome Statistically significant changes (p<0.01) are highlighted in red (upregulated). B) Volcano plot of mRNA data after annotation with the mouse reference genome are shown. Statistically significant changes (p<0.01) are highlighted in red (upregulated) or blue (downregulated).