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Abstract
The lead formulation of BBB-crossing lipid nanoparticles demonstrates substantially higher mRNA delivery efficiency into the brain than FDA-approved lipid nanoparticles.
- A total of 72 BBB-crossing lipids were designed and synthesized to enhance mRNA delivery to the brain.
- The novel lipid nanoparticles successfully transfect neurons and astrocytes across the entire brain in mouse models after intravenous injections.
- These nanoparticles are well tolerated across several dosage regimens.
- The ability to deliver mRNA to human brain samples was confirmed through ex vivo studies.
- This approach may provide a new platform for treating a variety of central nervous system diseases.
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