Lipid nanoparticle-based delivery of small interfering RNAs: New possibilities in the treatment of diverse diseases

Askarizadeh A., Vahdat-Lasemi F., KARAV S., Kesharwani P., Sahebkar A.

European Polymer Journal, vol.223, 2025 (SCI-Expanded) identifier

  • Publication Type: Article / Review
  • Volume: 223
  • Publication Date: 2025
  • Doi Number: 10.1016/j.eurpolymj.2024.113624
  • Journal Name: European Polymer Journal
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, PASCAL, Aerospace Database, Chemical Abstracts Core, Chimica, Communication Abstracts, Compendex, INSPEC, Metadex, Civil Engineering Abstracts
  • Keywords: Cancer, Drug delivery, Lipid-based nanoparticle, RNA interference, siRNA
  • Çanakkale Onsekiz Mart University Affiliated: Yes

Abstract

RNA interference (RNAi) is a well-known post-transcriptional gene-silencing mechanism that has garnered significant attention as a potentially powerful therapeutic procedure for combating recalcitrant diseases. Small interfering RNA (siRNA) as an effective RNAi tool mediates gene silencing pathway by mRNA degradation in cells and presents a unique strategy for the treatment of rebellious diseases. However, the low stability and suboptimal pharmacokinetic behavior of naked siRNAs have made it necessary to employ a delivery vehicle to protect siRNA against degradation and allow for its intracellular delivery. Among a plethora of available delivery platforms, lipid nanoparticles (LNPs) have received significant research attention and are currently recognized as the most advanced delivery system for RNA-based therapeutic agents. This is exemplified by the approval of Onpattro® for treating amyloidosis in the US and the European Union in 2018, as well as the development of COVID-19 mRNA vaccines. This review aims to provide a comprehensive evaluation of the potential effectiveness of lipid-based nanoparticles as a delivery system for siRNA in treating a wide array of diseases.