ABSTRACT

In recent years, a new application of RNA Nanotechnology was reported as for the modification of Extracellular Vesicles (EVs) (Figure 51.1). The orientation of arrow-shaped RNA was altered to control ligand-display on EV membranes for specific cell targeting or to regulate intracellular trafficking of siRNA/miRNA. Placing membrane-anchoring cholesterol at the arrow-tail results in display of RNA aptamer or folate on EV outer surface. Taking advantage of the RNA ligand for specific targeting and EVs for efficient membrane fusion, the resulting ligand-displaying EVs were competent for specific delivery of siRNA to cells without endosome trapping, which is critical for efficient RNAi therapy. Many of in vivo trials have demonstrated that the potency of this platform can be used to deliver anti-tumor siRNA and achieve tumor suppression. In this chapter, we summarized the current knowledge and techniques on using RNA nanotechnology in combination of EVs for gene therapy.