GSE99619
Liver DGE analysis of hemophilia mice (hFIX9-R29X) treated with ASOs inhibiting NMD
To evaluate the impact of ASO-mediated depletion of NMD factor Upf1 and Upf3b on the normal transcriptome, we performed digital gene expression analysison hFIX-R29X mouse liver treated with Upf3b-GalNAc-ASO. Overall design: hFIX-R29X mice 8-14 weeks of age (n=5-6, 2-3 female and 2-3 male mice per group) were treated every 5 days with 6 doses of DPBS, Control-GalNAc-ASO (15 or 20mg/kg), Upf1-GalNAc-ASO (20 mg/kg) and Upf3b-GalNAc-ASO (10 or 20 mg/kg). Animals were sacrificed 48 h after the last dose.