Exploiting endocytosis for siRNA delivery
The know-how in endocytosis positions the group at the leading edge of the studies of drug delivery systems. In cooperation with Alnylam Pharmaceuticals, Zerial’s group has been developing quantitative analytical methods to characterize the delivery of siRNAs to silence genes in cells in vitro and in mouse liver in vivo (Gilleron et al., 2013). Zerial’s group plans to extend this approach for discovering basic principles of tissue organization but also for translational research, i.e. 1) target identification in an organ of key importance for drug development and 2) development of new strategies for site-specific delivery of siRNAs and other biologically active macromolecules. For this, Zerial’s research can offer new solutions through bio-inspired delivery approaches.
Cutting edge technology specifically for liver delivery allows perturbations of a single component on the organ level and allows the specific knockdown of up to 10 genes in vivo. In this figure, we show a representative example of using this technology to knock down all 3 Rab5 isoforms in the mouse liver.