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There is a large excess capacity of ASGR1 in both engineered HEK cells and in vivo. (A) Comparison of uptake and activity of GalNAc-conjugated ASO in HEK 293 cell clones representing different levels of ASGR1 expression. (B) Activity of GalNAc-conjugated ASO in mouse liver under different levels of ASGR1 knockdown. Doses are expressed as mg ASO/kg body weight.
