Lipid nanoparticles improve activity of single-stranded siRNA and gapmer antisense oligonucleotides in animals

ACS Chem Biol. 2013 Jul 19;8(7):1402-6. doi: 10.1021/cb4001316. Epub 2013 May 2.

Abstract

We evaluated the abilities of an antisense oligonucleotide (ASO), a small interfering RNA (siRNA), and a single-stranded siRNA (ss-siRNA) to inhibit expression from the PTEN gene in mice when formulated identically with lipid nanoparticles (LNPs). Significantly greater reductions in levels of PTEN mRNA were observed for LNP-formulated agents compared to unformulated drugs when gene silencing was evaluated after a single dose in the livers of mice. An unformulated ss-siRNA modified with a metabolically stable phosphate mimic 5'-(E)-vinylphosphonate showed dose-dependent reduction of PTEN mRNA in mice, albeit at doses significantly higher than those observed for formulated ss-siRNA. These results demonstrate that LNPs can be used to deliver functional antisense and ss-siRNA therapeutics to the liver, indicating that progress in the field of siRNA delivery is transferable to other classes of nucleic acid-based drugs.

Publication types

  • Letter

MeSH terms

  • Animals
  • Dose-Response Relationship, Drug
  • Drug Delivery Systems
  • Gene Expression Regulation, Enzymologic / drug effects*
  • HeLa Cells
  • Humans
  • Inhibitory Concentration 50
  • Lipids / chemistry*
  • Liver / drug effects
  • Male
  • Mice
  • Mice, Inbred BALB C
  • Nanoparticles / chemistry*
  • Oligonucleotides, Antisense* / chemistry
  • Oligonucleotides, Antisense* / pharmacology
  • PTEN Phosphohydrolase / genetics*
  • RNA, Small Interfering* / chemistry
  • RNA, Small Interfering* / pharmacology

Substances

  • Lipids
  • Oligonucleotides, Antisense
  • RNA, Small Interfering
  • PTEN Phosphohydrolase