4.8 Article

Lipid nanoparticle-enabled gene editing in the lung via inhalation

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NATURE BIOTECHNOLOGY
卷 -, 期 -, 页码 -

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NATURE PORTFOLIO
DOI: 10.1038/s41587-023-01689-9

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A high-throughput platform has been developed to create lipid nanoparticles capable of delivering mRNA and CRISPR-Cas9 gene editing tools effectively to the lungs via intratracheal administration, expanding the potential clinical applications of gene editing and mRNA-based technologies.
The ability to create in vivo genomic medicines for tissues other than the liver has been impeded by difficulties in delivery. Using a high-throughput platform, we developed lipid nanoparticles that can effectively deliver mRNA and CRISPR-Cas9 gene editing tools to the lungs through intratracheal administration, expanding the potential clinical uses of gene editing and mRNA-based technologies.

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