Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides

Keith Henry Moss, Petya Popova, Sine Reker Hadrup, Kira Astakhova*, Maria Taskova

*Corresponding author for this work

Research output: Contribution to journalJournal articleResearchpeer-review

Abstract

Gene therapy is an exciting field that has the potential to address emerging scientific and therapeutic tasks. RNA-based gene therapy has made remarkable progress in recent decades. Nevertheless, efficient targeted delivery of RNA therapeutics is still a prerequisite for entering the clinics. In this review, we introduce current delivery methods for RNA gene therapeutics based on lipid nanoparticles (LNPs). We focus on the clinical appeal of recent RNA NPs and discuss existing challenges of fabrication and screening LNP candidates for effective translation into drugs of human metabolic diseases and cancer.
Original languageEnglish
JournalMolecular Pharmaceutics
Volume16
Issue number6
Pages (from-to)2265-2277
Number of pages13
ISSN1543-8384
DOIs
Publication statusPublished - 2019

Keywords

  • Gene therapy
  • RNA
  • Delivery
  • Lipid nanoparticle (LNP)
  • LNP fabrication
  • LNP screening

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