Gene delivery and editing in the front part of the eye using lipid nanoparticles carrying mRNA

IC injections of lipid nanoparticles (LNPs) resulted in robust protein expression and gene editing in the trabecular meshwork.

• LNP-mediated delivery of Cre-mRNA led to significant tdTomato expression in the angle and corneal endothelium of Ai9 mice.
• Localized protein expression from mCherry-mRNA LNPs in C57BL/6 mice peaked at 72 hours and declined over 120 hours.
• LNPs encapsulating Cas9 mRNA with sgAi9 achieved up to 14.3% editing efficiency for in vivo gene editing in Ai9 mice.
• tdTomato expression was induced in the iridocorneal angle, demonstrating the potential for LNPs in gene editing applications.
• No ocular toxicity was observed with the intracameral administration of LNPs, suggesting safety for this delivery method.

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