Fig. 5: Effect of defective cholesterol trafficking on eLNP-mediated gene transfection.

LNPs and eLNPs were compared for their ability to deliver mRNA to a wild-type (WT) and NPC1 knockout (NPC1−/−) mouse embryonic fibroblasts (200 ng mRNA per well), and b patient-derived NPC1, and c NPC2 loss-of-function fibroblasts. All experiments were conducted with n = 3; mean ± SD. (*p ≤ 0.05, **p ≤ 0.01, ***p ≤ 0.001. Significance was determined using multiple t-test.) Source data are provided as a Source Data file.