Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides

Mol Pharm. 2019 Jun 3;16(6):2265-2277. doi: 10.1021/acs.molpharmaceut.8b01290. Epub 2019 May 16.

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.

Keywords: LNP fabrication; LNP screening; RNA; delivery; gene therapy; lipid nanoparticle (LNP).

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Genetic Therapy / methods
  • Humans
  • Lipids / chemistry*
  • Nanoparticles / chemistry*
  • Oligonucleotides / chemistry*
  • RNA / chemistry*

Substances

  • Lipids
  • Oligonucleotides
  • RNA