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Updated: May 23, 2025

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA
Published on: February 1, 2019
Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing in vivo
Xuexiang Han1,2,3, Ying Xu4,5,6,3, Adele Ricciardi1,7,3
1Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA.
Abstract:
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 in vivo. Our study highlights the utility of this synthetic approach and the generality of this platform for potent in vivo gene editing.
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