How to Tackle the Challenge of siRNA Delivery with Sequence-Defined Oligoamino Amides

Reinhard, Sören and Wagner, Ernst (2017): How to Tackle the Challenge of siRNA Delivery with Sequence-Defined Oligoamino Amides. In: Macromolecular Bioscience, Vol. 17, No. 1, 1600152

Abstract

RNA interference (RNAi) as a mechanism of gene regulation provides exciting opportunities for medical applications. Synthetic small interfering RNA (siRNA) triggers the knockdown of complementary mRNA sequences in a catalytic fashion and has to be delivered into the cytosol of the targeted cells. The design of adequate carrier systems to overcome multiple extracellular and intracellular roadblocks within the delivery process has utmost importance. Cationic polymers form polyplexes through electrostatic interaction with negatively charged nucleic acids and present a promising class of carriers. Issues of polycations regarding toxicity, heterogeneity, and polydispersity can be overcome by solid-phase-assisted synthesis of sequence-defined cationic oligomers. These medium-sized highly versatile nucleic acid carriers display low cytotoxicity and can be modified and tailored in multiple ways to meet specific requirements of nucleic acid binding, polyplex size, shielding, targeting, and intracellular release of the cargo. In this way, sequence-defined cationic oligomers can mimic the dynamic and bioresponsive behavior of viruses.

• Item Type: Journal article
• Faculties: Chemistry and Pharmacy > Department of Pharmacy
• Research Centers: Nanosystems Initiative Munich (NIM)
• Subjects: 500 Science > 540 Chemistry; 500 Science > 530 Physics
• ISSN: 1616-5187
• Language: English