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Abstract
Lipid nanoparticles designed with peptide ionizable lipids enable organ-selective mRNA delivery.
- Peptide ionizable lipids facilitate the assembly of lipid nanoparticles with specific targeting to organs such as the lungs, liver, spleen, thymus, and bone.
- Incorporating artificial ionizable and natural amino acids into these lipids results in enhanced tissue-specific delivery.
- Lipid nanoparticles targeting the liver demonstrate efficacy and safety levels comparable to FDA-approved formulations.
- This approach allows for the efficient co-delivery of mRNA and engineered guide RNA for prime editing in liver and lung tissues.
- A predictable strategy for designing these lipid nanoparticles may improve mRNA-based gene editing therapies.
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