Lipid nanoparticles for targeted siRNA delivery – going from bench to bedside

Abstract

This review covers the basic aspects of small interfering RNA delivery by lipid nano-particles (LNPs) and elaborates on the current status of clinical trials for these systems. We briefly describe the roles of all LNP components and possible strategies for their improvement. We also focus on the current clinical trials using LNP-formulated RNA and the possible outcomes for therapy in the near future. Also, we present a critical analysis of selected clinical trials that reveals the common logic behind target selection. We address this review to a wide audience, especially to medical doctors who are interested in the application of RNA interference–based treatment platforms. We anticipate that this review may spark interest in this particular audience and generate new ideas in target selection for the disorders they are dealing with.

Keywords:

• RNA therapeutics
• siRNA
• mRNA
• lipid nanoparticle
• targeted delivery
• clinical trial

Acknowledgments

This work was supported by the Russian Scientific Foundation (grant no 14-34-00017). The authors are grateful to Dominique Leboeuf for critical reading of the manuscript.

Disclosure

The authors report no conflicts of interest in this work.