GSPT1 and hemophilia: The amount of hFIX protein detected in male hemophilia mice with either the combination of Upf3b-GalNAc-ASO and Gspt1-GalNAc-ASO, or the combination of Upf3b-GalNAc-ASO and geneticin, although reliably detected, remained lower than the threshold 1% of hFIX-WT abundance (Fig. 7d).