Home Products Cited in Publications Worldwide Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing in vivo
Han, Xuexiang; Xu, Ying; Ricciardi, Adele; Xu, Junchao; Palanki, Rohan; Chowdhary, Vivek; Xue, Lulu; Gong, Ningqiang; Alameh, Mohamad-Gabriel; Pereanteau, William
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.