Identification of potent siRNA targeting complement C5 and its robust activity in pre-clinical models of myasthenia gravis and collagen-induced arthritis

Lipid nanoparticle-targeted mRNA formulation as a treatment for ornithine-transcarbamylase deficiency model mice

“…In this study, we used our biodegradable ionizable-based lipid nanoparticles (LNP) 21 , 22 , 23 , 24 , 25 to formulate engineered mRNA encoding h OTC in the LNP (h OTC -mRNA/LNP) for delivery to hepatocytes to treat OTCD. The three-dimensional structure of the hOTC protein produced in cells treated with h OTC -mRNA/LNP was determined, and the localization of the hOTC protein to mitochondria was demonstrated.…”

Understanding the Manufacturing Process of Lipid Nanoparticles for mRNA Delivery Using Machine Learning

“…These human cell lines are derived from liver tissue, where intravenously administrated LNPs are mainly delivered, as previously reported. 54,55) The data of the protein expression levels in cells treated with the four LNP formulations were analyzed using one-way ANOVA followed by Tukey's multiple comparisons test. The results showed that there were statistically significant differences in the protein expression levels in both cell types for each particle size LNPs with 100 ng/mL mRNA (Figs.…”

Myasthenia gravis: the future is here

“…Ongoing research efforts continue to explore the complement system as a target for MG treatment. For example, a siRNA that inhibits hepatic synthesis of C5 has shown promise in reducing the severity of EAMG ( 138 , 139 ). Additionally, C7 has been effectively inhibited in EAMG models, further highlighting the potential of complement system targeting in MG therapeutics ( 30 ).…”