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. 2020 Jul 24;6(30):eabb4429. doi: 10.1126/sciadv.abb4429

Fig. 3. NT-LNP facilitates the delivery of Tau-ASO into the mouse brain and for gene knockdown in both mRNA and protein levels.

Fig. 3

(A) Chemical structure of 306-O12B-3, NT1-O14B, and schematic illustration of the doped NT-lipidoid Tau-ASO formulation for brain delivery. (B) GFP silencing efficiency of HEK-GFP cells treated with or without ASO/NT-LNP complexes. The NT1-O14B LNPs alone showed no silencing efficacy, while doping NT1-lipidoid into 306-O12B-3 LNPs led to successful gene silencing in vitro. *P < 0.01 versus all other samples in the same group. (C) Tau-ASOs formulated with NT1-O14B doped with different ratios of 306-O12B-3, saline, or scrambled Tau-ASO-LNPs were intravenously injected into C57BL/6J mice (n = 6 per group) via the tail vein, and the brain was analyzed for total tau mRNA levels. Graphical data are represented as box and whisker plots with individual points overlaid, where error bars represent maximum and minimum values and the boxed line represents the median, *P < 0.05 or **P < 0.001. (D) Total tau protein levels of the NT1-O14B/306-O12B-3 = 3:7 group, comparing to that of saline or scrambled Tau-ASO, **P < 0.001. One-way ANOVA, Sidak post hoc analysis.