mRNA-based gene editing therapeutics offer the potential to permanently cure diseases but are hindered by suboptimal delivery platforms. Here, we devise a robust combinatorial chemistry for plug-and-play assembly of diverse biodegradable ionizable lipids and identify a lead candidate that produces superior lipid nanoparticles for various gene editing tools delivery . Our study highlights the utility of this synthetic approach and the generality of this platform for potent gene editing.
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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11888472 | PMC |
| http://dx.doi.org/10.1101/2025.02.25.640222 | DOI Listing |
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