Bone Abstracts

Since its discovery <20 years ago, RNA interference (RNAi) has proven to be a powerful tool for the downregulation of any target gene of choice. This also offers the use of RNAi-inducing small interfering RNAs (siRNAs) in a therapeutic setting, for the knockdown of pathologically overexpressed genes in various pathologies. Beyond siRNAs, this concept has been extended towards microRNAs (miRNAs) and their inhibitors.

While the target cell provides the RNAi machinery, the delivery of the siRNA is still the major bottleneck in therapeutic RNAi use. Non-viral strategies include, among others, chemical siRNA modification and its coupling to fusion partners for targeted delivery as well as various approaches based on nanoparticle formulation.

This presentation highlights the major issues in siRNA application and gives examples for solutions towards the goal of developing RNAi therapeutics. Beyond siRNA conjugates, the use of liposomal or polymeric nanoparticles for delivery is discussed. Preclinical and clinical examples are given.