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Showing 1 to 4 of 4 for “"LNP delivery"”.
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Design, synthesis, and biological evaluation of diketopiperazine based ionizable lipids for the in vivo delivery of messenger RNA
… of specific proteins. The successful delivery of messenger RNA (mRNA) to target cells in the body could accomplish this goal, but serious limitations with its systemic delivery must still be overcome. Recently, lipid nanoparticles (LNPs) have shown promise for mRNA delivery in vivo, …
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Development and mechanistic analysis of in vivo liposomal nanoparticle delivery of siRNA and mRNA
… RNA to cells in vivo. Liposomal nanoparticles (LNPs) have shown some success, but their design is limited by both a lack of information concerning the optimal lipid composition and insufficient data regarding the LNPs' interaction with their environment. Further elucidation of the physical …
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RNA Therapeutics for Fibrolamellar Hepatocellular Carcinoma
… receptor (ASGR), allowing targeted delivery to hepatocytes. I hypothesized that ASGR might still be sufficiently expressed in FLC tumors to deliver siRNAs to cells in vivo. I demonstrated that indeed ASGR is still expressed across different stages of the FLC in patient samples and …
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Tie-ing the endothelium: development of an mRNA-based nanotherapeutic to restore microvascular barrier function for the treatment of sepsis-induced multiple organ failure
… of cellular pathways. However, intracellular delivery of mRNA remains a hurdle due to its large size, polyanionic nature, and susceptibility to degradation. Lipid nanoparticles (LNPs) containing ionizable cationic lipids emerged as the most clinically advanced platform for mRNA delivery, but …