Lipid-like materials for low-dose, in vivo gene silencing

Proc Natl Acad Sci U S A. 2010 Feb 2;107(5):1864-9. doi: 10.1073/pnas.0910603106. Epub 2010 Jan 11.

Abstract

Significant effort has been applied to discover and develop vehicles which can guide small interfering RNAs (siRNA) through the many barriers guarding the interior of target cells. While studies have demonstrated the potential of gene silencing in vivo, improvements in delivery efficacy are required to fulfill the broadest potential of RNA interference therapeutics. Through the combinatorial synthesis and screening of a different class of materials, a formulation has been identified that enables siRNA-directed liver gene silencing in mice at doses below 0.01 mg/kg. This formulation was also shown to specifically inhibit expression of five hepatic genes simultaneously, after a single injection. The potential of this formulation was further validated in nonhuman primates, where high levels of knockdown of the clinically relevant gene transthyretin was observed at doses as low as 0.03 mg/kg. To our knowledge, this formulation facilitates gene silencing at orders-of-magnitude lower doses than required by any previously described siRNA liver delivery system.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Biocompatible Materials / chemical synthesis
  • Biocompatible Materials / chemistry*
  • Drug Delivery Systems
  • Factor VII / antagonists & inhibitors
  • Factor VII / genetics
  • Gene Silencing*
  • HeLa Cells
  • Hepatocytes / metabolism
  • Humans
  • Lipids / chemical synthesis
  • Lipids / chemistry*
  • Macaca fascicularis
  • Mice
  • Mice, Inbred C57BL
  • Molecular Structure
  • RNA Interference
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / genetics*

Substances

  • Biocompatible Materials
  • Lipids
  • RNA, Small Interfering
  • Factor VII