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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References
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Gillmore, J. D., Maitland, M. L. & Lebwohl, D. CRISPR–Cas9 in vivo gene editing for transthyretin amyloidosis. Reply. N. Engl. J. Med. 385, 1722–1723 (2021). This paper reports LNP-enabled CRISPR–Cas9 gene editing in a human trial.
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This is a summary of: Li, B. et al. Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing. Nat. Biotechnol. https://doi.org/10.1038/s41587-023-01679-x (2023).
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Lipid nanoparticle-enabled gene editing in the lung via inhalation. Nat Biotechnol (2023). https://doi.org/10.1038/s41587-023-01689-9
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DOI: https://doi.org/10.1038/s41587-023-01689-9
