Targeted systemic delivery of a therapeutic siRNA with a multifunctional carrier controls tumor proliferation in mice

Mol Pharm. 2009 May-Jun;6(3):738-46. doi: 10.1021/mp800192d.

Abstract

In this study, novel peptide-targeted delivery systems were developed for systemic and targeted delivery of therapeutic siRNA based on a multifunctional carrier, (1-aminoethyl)iminobis[N-(oleicylcysteinylhistinyl-1-aminoethyl)propionamide] (EHCO), which showed pH-sensitive amphiphilic cell membrane disruption. EHCO formed stable nanoparticles with siRNA. Targeted siRNA delivery systems were readily formed by surface modification of the nanoparticles. PEGylation of the siRNA/EHCO nanoparticles significantly reduced nonspecific cell uptake. The incorporation of a bombesin peptide or RGD peptide via a PEG spacer resulted in receptor-mediated cellular uptake and high gene silencing efficiency in U87 cells. Fluorescence confocal microscopic studies demonstrated that EHCO/siRNA nanoparticles and PEG modified EHCO/siRNA nanoparticles were able to facilitate endosomal escape of the siRNA delivery systems. Systemic administration of a therapeutic anti-HIF-1alpha siRNA with the peptide-targeted delivery systems resulted in significant tumor growth inhibition than a nontargeted delivery system or free siRNA via intravenous injection in nude mice bearing human glioma U87 xenografts. The results indicate a great promise of the multifunctional carrier EHCO for systemic and targeted delivery of therapeutic siRNA to treat human diseases with RNAi.

MeSH terms

  • Animals
  • Cell Proliferation / drug effects
  • Flow Cytometry
  • Gene Silencing / physiology
  • Glioma / drug therapy*
  • Humans
  • Hypoxia-Inducible Factor 1, alpha Subunit / genetics
  • Injections, Intravenous
  • Mice
  • Mice, Nude
  • Microscopy, Confocal
  • Microscopy, Fluorescence
  • Molecular Structure
  • Nanoparticles / administration & dosage
  • Nanoparticles / chemistry
  • Oligopeptides / chemistry
  • RNA, Small Interfering / administration & dosage
  • RNA, Small Interfering / chemistry*
  • RNA, Small Interfering / pharmacology*
  • RNA, Small Interfering / therapeutic use*
  • Transplantation, Heterologous

Substances

  • Hypoxia-Inducible Factor 1, alpha Subunit
  • Oligopeptides
  • RNA, Small Interfering
  • arginyl-glycyl-aspartic acid