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Abstract
A single intrathecal injection of TD5 brain-targeting lipid nanoparticles led to GFP expression in 29.6% of neurons and 38.1% of astrocytes across the brain.
- TD5 brain-targeting lipid nanoparticles (BLNPs) outperformed FDA-approved formulations in mRNA delivery to the central nervous system.
- In an Ai14 mouse model, TD5 BLNPs induced tdTomato expression in approximately 30% of neurons and 40% of astrocytes.
- Delivery of Cas9 mRNA/sgRNA complex via TD5 BLNPs enabled effective genome editing in the brain.
- TD5 BLNPs demonstrated safety profiles comparable to existing lipid nanoparticle formulations, suggesting good biocompatibility.
- This formulation may provide a promising strategy for mRNA delivery in treating central nervous system diseases.
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