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. Author manuscript; available in PMC: 2024 Apr 18.
Published in final edited form as: ACS Nano. 2023 Jul 17;17(14):13594–13610. doi: 10.1021/acsnano.3c02268

Fig. 5 |. C3 LNP-mediated in utero mRNA delivery to the NHP brain and ex vivo performance in pediatric biological fluids and brain tissue.

Fig. 5 |

(A) Experimental scheme depicting ultrasound guided ICV injection of C3 LNP.GFP in a 0.61G Macaca fascicularis. (B) Ultrasound images (red arrow points to needle) pre- and post-ICV injection of C3.GFP LNPs in fetal macaque. (C) GFP immunohistochemistry on brain sections from C3 LNP.GFP-treated and negative control animals. Scale bars: 100 μm (10X). (D) Size and PDI measurements of C3.MPS LNPs incubated in PBS, human CSF, or human serum. **** p < 0.0001 by one-way ANOVA with Tukey’s multiple comparisons test. (E) Size measurements of C3.MPS LNPs incubated in human CSF over a 7-day time course. (F) GFP positivity in two patient-derived primary cell lines enriched for neurons after C3.GFP LNP treatment. (G) Viability after C3.GFP LNP treatment in both patient-derived cell lines. (H) NGS results in patient-derived precision cut brain slices treated with C3.MPS LNPs normalized to control (PBS). Outliers were detected using Grubbs’ test and removed from analysis; minimum n = 3 per treatment group; error bars represent SEM.