Cell type-specific delivery of short interfering RNAs by dye-functionalised theranostic nanoparticles

Nat Commun. 2014 Dec 3:5:5565. doi: 10.1038/ncomms6565.

Abstract

Efficient delivery of short interfering RNAs reflects a prerequisite for the development of RNA interference therapeutics. Here, we describe highly specific nanoparticles, based on near infrared fluorescent polymethine dye-derived targeting moieties coupled to biodegradable polymers. The fluorescent dye, even when coupled to a nanoparticle, mimics a ligand for hepatic parenchymal uptake transporters resulting in hepatobiliary clearance of approximately 95% of the dye within 45 min. Body distribution, hepatocyte uptake and excretion into bile of the dye itself, or dye-coupled nanoparticles can be tracked by intravital microscopy or even non-invasively by multispectral optoacoustic tomography. Efficacy of delivery is demonstrated in vivo using 3-hydroxy-3-methyl-glutaryl-CoA reductase siRNA as an active payload resulting in a reduction of plasma cholesterol levels if siRNA was formulated into dye-functionalised nanoparticles. This suggests that organ-selective uptake of a near infrared dye can be efficiently transferred to theranostic nanoparticles allowing novel possibilities for personalised silencing of disease-associated genes.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cholesterol / blood
  • Drug Delivery Systems
  • Fluorescent Dyes / metabolism*
  • HEK293 Cells
  • Hepatocytes / metabolism*
  • Humans
  • Hydroxymethylglutaryl CoA Reductases / genetics
  • Indoles / metabolism*
  • Nanoparticles / metabolism*
  • RNA Interference
  • RNA, Small Interfering / administration & dosage*
  • Rats

Substances

  • Fluorescent Dyes
  • Indoles
  • RNA, Small Interfering
  • VPM chloride
  • Cholesterol
  • Hydroxymethylglutaryl CoA Reductases