RNA and DNA nanoparticles for triggering RNA interference

Ziad El Tannir ,Kirill A Afonin ,Bruce A Shapiro

doi:10.14800/rd.724

Abstract

Control over the delivery of different functionalities and their synchronized activation in vivo is a challenging undertaking that requires careful design and implementation. The goal of the research highlighted herein was to develop a platform allowing the simultaneous activation of multiple RNA interference pathways and other functionalities inside cells. Our team has developed several RNA, RNA/DNA and DNA/RNA nanoparticles able to successfully complete such tasks. The reported designs can potentially be used to target myriad of different diseases.

Highlights

We introduced a technique that allows the conditional activation of RNA interference (RNAi) in vivo [16]
RNA interference (RNAi) is the biological process of specific gene silencing through a natural cellular post-transcriptional regulation process that involves short double stranded-RNAs [9,10,11]
The precise controlled delivery of various RNAi-based therapeutics could be achieved by building programmable RNA scaffolds that can be further functionalized and assembled into RNA nanoparticles of various shapes and compositions [2,3,14,15]

Summary

Introduction

We introduced a technique that allows the conditional activation of RNAi in vivo [16]. The goal of the research highlighted was to develop a platform allowing the simultaneous activation of multiple RNA interference pathways and other functionalities inside cells.

Results

Conclusion