1Pharmaceutical Biotechnology, Center for System-Based Drug Research, and 2Center for Nanoscience (CeNS), Ludwig Maximilians University, Munich, D-81377, Germany
Precise sequence-defined cationic oligomers were designed for siRNA delivery. Solid-phase assisted synthesis enables the generation of nanosized multifunctional siRNA shuttles. The endosomal protonatable 1,2-diaminoethane motif, cysteines and/or tyrosine trimers or dioleic/dilinolic acids were found as critical for nanoparticle stabilization and functional siRNA delivery. Precise PEG-ligands confer shielding and targeting properties in vitro and in vivo.