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. 2023 May 15;14:2776. doi: 10.1038/s41467-023-37465-1

Fig. 2. GalNAc-Lipid optimization in LDLR-deficient mouse models.

Fig. 2

a GalNAc-Lipid GL6 comprising a PEG spacer and 1,2-di-O-octadecyl-sn-glyceryl lipid anchor; b titration of the surface density of GalNAc-Lipid demonstrated a low density near 0.05 mol % of GalNAc-Lipid optimally rescues liver editing in female 8–10 week old Ldlr –/– mice while preserving editing in wild type (WT) mice at an RNA dose of 0.1 mg/kg (Table S1, entries 9–14); c LNPs constituted with 0.05 mol % GL6 maintained Angptl3 base editing in female WT and Ldlr +/– mice and rescued base editing in Ldlr –/– mice in vivo at 0.25 mg/kg; d demonstration of near-identical dose response of liver Angptl3 editing using the optimized GalNAc-LNPs (constituted with 0.05 mol % GL6) in three genotypes: WT, Ldlr +/–, and Ldlr –/–. Data are presented as mean values + /- standard deviation, and individual data points for each animal are displayed (n = 5). Source data are provided as a Source Data file.