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Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing in vivo
Easy-to-build biodegradable lipids for strong mRNA delivery and gene editing in living animals
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Abstract
Screening 500 ionizable lipids led to the identification of a potent candidate for mRNA delivery.
- A combinatorial chemistry approach enabled the assembly of diverse biodegradable lipids for gene delivery.
- Machine learning facilitated the understanding of structure-activity relationships for effective mRNA delivery.
- The lead lipid candidate outperformed both literature and industry benchmark lipid nanoparticles in mice.
- Favorable physicochemical properties of the lipid nanoparticles may enhance their delivery performance.
- The synthetic method demonstrates potential for broad application in in vivo gene editing.
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