Cell penetrating peptide-mediated systemic siRNA delivery to the liver

Int J Pharm. 2011 Oct 31;419(1-2):308-13. doi: 10.1016/j.ijpharm.2011.07.038. Epub 2011 Jul 30.

Abstract

The cell-penetrating peptide (CPP) is one of the most attractive tools for efficiently delivering biomolecules to a target organelle. Here, we describe the use of octaarginine (R8)-modified lipid nanoparticles for the efficient and targeted in vivo delivery of siRNA to the liver. In this study, SR-BI (a scavenger receptor class B, member 1) was targeted by this nanoparticle. Our results demonstrate that R8-modified lipid nanoparticles can be used for the efficient and targeted delivery of liver siRNA to induce the specific knock-down of an endogenous gene with minimum liver toxicity and immune response, and that this CPP based technology holds considerable promise for further in vivo biological applications of siRNA.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • CD36 Antigens / genetics
  • Cell-Penetrating Peptides / chemistry*
  • Female
  • Gene Knockdown Techniques
  • Gene Transfer Techniques
  • Lipids / chemistry
  • Liver / metabolism*
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred ICR
  • Nanoparticles*
  • Oligopeptides / chemistry
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / toxicity

Substances

  • CD36 Antigens
  • Cell-Penetrating Peptides
  • Lipids
  • Oligopeptides
  • RNA, Small Interfering
  • octaarginine