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Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
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Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
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Journal Article
Combinatorial design of nanoparticles for pulmonary mRNA delivery and genome editing
2023
Overview
The expanding applications of nonviral genomic medicines in the lung remain restricted by delivery challenges. Here, leveraging a high-throughput platform, we synthesize and screen a combinatorial library of biodegradable ionizable lipids to build inhalable delivery vehicles for messenger RNA and CRISPR–Cas9 gene editors. Lead lipid nanoparticles are amenable for repeated intratracheal dosing and could achieve efficient gene editing in lung epithelium, providing avenues for gene therapy of congenital lung diseases.
A high-throughput screen improves lipid nanoparticle delivery of gene editors in the lung.
