Octaarginine-modified multifunctional envelope-type nanoparticles for gene delivery

Gene Ther. 2007 Apr;14(8):682-9. doi: 10.1038/sj.gt.3302910. Epub 2007 Feb 1.

Abstract

This study describes a multifunctional envelope-type nano device (MEND) that mimics an envelope-type virus based on a novel packaging strategy. MEND particles contain a DNA core packaged into a lipid envelope modified with an octaarginine peptide. The peptide mediates internalization via macropinocytosis, which avoids lysosomal degradation. MEND-mediated transfection of a luciferase expression plasmid achieved comparable efficiency to adenovirus-mediated transfection, with lower associated cytotoxicity. Furthermore, topical application of MEND particles containing constitutively active bone morphogenetic protein (BMP) type IA receptor (caBmpr1a) gene had a significant impact on hair growth in vivo. These data demonstrate that MEND is a promising non-viral gene delivery system that may provide superior results to existing non-viral gene delivery technologies.

Publication types

  • Comparative Study
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenoviridae / genetics
  • Animals
  • Cell Line
  • Gene Expression
  • Genetic Engineering
  • Genetic Therapy / methods*
  • Humans
  • Luciferases / analysis
  • Luciferases / genetics
  • Mice
  • Mice, Mutant Strains
  • Microscopy, Confocal
  • Nanoparticles
  • Oligopeptides / genetics*
  • Skin / metabolism
  • Skin / virology
  • Transfection / methods*
  • beta-Galactosidase / analysis
  • beta-Galactosidase / genetics

Substances

  • Oligopeptides
  • octaarginine
  • Luciferases
  • beta-Galactosidase