mRNA-loaded lipid nanocapsules for ocular delivery
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Date
2025-07-09
Authors
O'Reilly, Tanibet Alba
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Abstract
Eye disorders affect the quality of life of humans. Recent advances suggest that gene therapies are a promising option for treating such conditions, due to the accessibility of the eyes, which allows for localized treatment. Nevertheless, biological barriers within the eye complicate the efficient delivery of oligonucleotides. One promising approach involves delivering mRNA to restore the expression of a deficient protein associated with a specific disease. Nanocarriers can be employed to shield the unstable mRNA from degradation. This study aimed to evaluate the potential of mRNA-loaded lipid nanocapsules to mediate gene expression in human retinal cells and mice, using Enhanced Green Fluorescent Protein (EGFP) mRNA as a model. The LNCs were successfully prepared via the phase inversion technique, resulting in nanoparticles with an average size of 80 nm, a polydispersity index below 0.1, and a positive zeta potential of +10 mV. The particles were purified by size exclusion chromatography, and the encapsulation efficiency and the drug loading obtained were 59% and 0.002%, respectively. The nanoparticle suspension was tested in human retinal pigmented epithelium cells (ARPE-19), showing no cytotoxicity, particularly at concentrations below 1 mg/mL. The in vivo experiments showed promising results regarding the in vivo transfection of retinal cells. These findings support the potential of this LNC formulation as an effective platform for mRNA delivery to the eye.
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Keywords
Lipid nanocapsules (LNC), mRNA delivery, Ocular delivery