Abstract

During the last decade, an increased amount of oligonucleotides have been developed as promising therapeutic agents for treatment of diseases. siRNA/shRNA is one promising example of therapeutic oligonucleotides which can inhibit target gene expression by degrading mRNA in a highly sequence-specific manner. Although, siRNA shows potential therapeutic properties, the specific delivery remains a major barrier for clinical development of siRNA-based drugs. In this chatper, we provide an overview of current progress in the field of siRNA/shRNA delivery and discuss recent patents and technical advances in the development of efficient small RNA delivery vehicles including strategies to enhance their pharmacokinetic properties, to promote their cellular uptake, and to foster corresponding clinical trials.