William Cantley

TL;DR: The best-performing lipid recovered after screening (DLin-KC2-DMA) was formulated and characterized in SNALP and demonstrated to have in vivo activity at siRNA doses as low as 0.01 mg/kg in rodents and 0.1 mg/ kg in nonhuman primates, a substantial improvement over previous reports of in vivo endogenous hepatic gene silencing.

TL;DR: Functional evidence for a novel regulatory pathway for lipoprotein metabolism is provided and it is suggested that modulation of this pathway may alter risk for MI in humans.

TL;DR: A formulation has been identified that enables siRNA-directed liver gene silencing in mice at doses below 0.01 mg/kg and was shown to specifically inhibit expression of five hepatic genes simultaneously, after a single injection.

TL;DR: A targeting approach using an exogenous ligand containing a multivalent N-acetylgalactosamine (GalNAc)-cluster, which binds with high affinity to the asialoglycoprotein receptor (ASGPR) expressed on hepatocytes appears to be highly effective for the delivery of iLNPs to liver.

TL;DR: An optimized lipid nanoparticle and a CCR2-silencing short interfering RNA that, when administered systemically in mice, show rapid blood clearance, accumulate in spleen and bone marrow, and localize to monocytes prevent their accumulation in sites of inflammation.