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Abstract
One lipid nanoparticle (LNP1) efficiently delivered mRNA to specific brain cells in fetal models.
- Lipid nanoparticles are effective for delivering messenger RNA for base editing but struggle with targeting the central nervous system.
- LNP1 was identified as capable of delivering mRNA to endothelial cells, microglia, and neurons in the fetal cortex.
- Application of LNP1 in a mouse model for spinal muscular atrophy enabled base editing in the liver and cortex.
- Low levels of editing were observed in central nervous system organs, yet fetal administration led to modestly increased survival in SMA mice.
- Findings suggest that fetal biology may play a role in enhancing mRNA delivery for potential central nervous system therapies.
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