Eugenio Fava

TL;DR: It is estimated that escape of siRNAs from endosomes into the cytosol occurs at low efficiency (1–2%) and only during a limited window of time when the LNPs reside in a specific compartment sharing early and late endosomal characteristics.

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: It is shown that a high number of kinases are involved in endocytosis, and that each endocytic route is regulated by a specific kinase subset, suggesting coordinate regulation.

TL;DR: A new strategy to phenotypically profile the human genome with respect to transferrin (TF) and epidermal growth factor (EGF) endocytosis is developed by combining RNA interference, automated high-resolution confocal microscopy, quantitative multiparametric image analysis and high-performance computing.

TL;DR: It is demonstrated that siRNA activity is primarily enhanced by favouring the incorporation of the intended antisense strand during RNA-induced silencing complex (RISC) loading by modulation of siRNA thermodynamic asymmetry and engineering of siRNAs 3′-overhangs.