Overcoming the blood-brain barrier using central nervous system-accessing lipid nanoparticles for enhanced mRNA therapeutics.
This work designed, synthesized, and tested a series of ionizable lipids and formulated them into CNS-accessing lipid nanoparticles (CA LNPs) to deliver mRNA, and the lead candidate, CA2d LNP, demonstrated efficient mRNA delivery across the blood-brain barrier following intravenous injection.