• Strategies for target delivery of RNAi: Review Abstract Article
  • sadaf safaei,1,* Hamid Mir Mohammad Sadeghi,2
    1. Department of pharmaceutical biotechnology, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical Sciences, Isfahan, Iran
    2. Department of pharmaceutical biotechnology, School of Pharmacy and Pharmaceutical Sciences, Isfahan University of Medical Sciences, Isfahan, Iran


  • Introduction: In last decades, RNA interference (RNAi) technology has become a powerful tool for clinical approaches. These oligonucleotides, targeted mRNA with high specificity and efficacy and induce degradation of the targeted mRNA by the RISC machinery. Inhibition the expression of protein is the key for the purpose of treatment of diseases. The major challenge for RNAi application is enhance its stability and bioactivity. PEGylated lipid-based delivery system plays an important role for targeting delivery of nucleic acids. In this review, we discuss PEGylated lipid-based RNAi delivery systems and strategies to overcome technical barriers for target delivery of RNAi.
  • Methods: Many strategies were developed to overcome technical barriers for target delivery of RNAi. Viral vectors, such as adenovirus or lentivirus, chemical methods, physical methods (hydrodynamic injection, particle bombardment, and electroporation and polymer, lipid-based delivery systems, Self-assembled lipid-polymer hybrid NPs, these are strategies, which explain the difficulty of this approach to enhance endosome escaping efficiency and target delivery of RNAi.
  • Results: Viral vectors are efficient in gene delivery, but they have some contraindication due to many drawbacks such as insertional mutagenesis, immunogenic and inflammatory responses. Non-viral vectors, including Lipid-based nanoparticles were developed as a promising platform to enhance target delivery of RNAi. PEGylated lipid-based delivery system plays an important role for target delivery of RNAi. Key advantages of PEGylated lipid-based delivery system is increase the circulation time, low immunogenicity, ability to deliver large size genetic materials, cost-effective manufacturing.
  • Conclusion: In recent years, variety of lipid-based nanoparticles were developed as a promising platform to enhance target delivery of RNAi. PEGylated lipid-based delivery system plays an important role for targeting delivery of nucleic acids due to increase the circulation time, low toxicity, biocompatibility, and facilitating scaled up. RNAi-PEG conjugates, suggesting that they can be potential promising platform for clinical approaches.
  • Keywords: RNAi, RISC machinery, PEGylating