Mechanisms and optimization of in vivo delivery of lipophilic siRNAs

Nat Biotechnol. 2007 Oct;25(10):1149-57. doi: 10.1038/nbt1339. Epub 2007 Sep 16.

Abstract

Cholesterol-conjugated siRNAs can silence gene expression in vivo. Here we synthesize a variety of lipophilic siRNAs and use them to elucidate the requirements for siRNA delivery in vivo. We show that conjugation to bile acids and long-chain fatty acids, in addition to cholesterol, mediates siRNA uptake into cells and gene silencing in vivo. Efficient and selective uptake of these siRNA conjugates depends on interactions with lipoprotein particles, lipoprotein receptors and transmembrane proteins. High-density lipoprotein (HDL) directs siRNA delivery into liver, gut, kidney and steroidogenic organs, whereas low-density lipoprotein (LDL) targets siRNA primarily to the liver. LDL-receptor expression is essential for siRNA delivery by LDL particles, and SR-BI receptor expression is required for uptake of HDL-bound siRNAs. Cellular uptake also requires the mammalian homolog of the Caenorhabditis elegans transmembrane protein Sid1. Our results demonstrate that conjugation to lipophilic molecules enables effective siRNA uptake through a common mechanism that can be exploited to optimize therapeutic siRNA delivery.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Apolipoproteins B / genetics
  • Apolipoproteins B / metabolism
  • Cholesterol
  • Cricetinae
  • Gene Transfer Techniques*
  • Lipoproteins, HDL
  • Lipoproteins, LDL / genetics
  • Lipoproteins, LDL / metabolism
  • Mesocricetus
  • Mice
  • RNA Interference*
  • RNA, Small Interfering / genetics*
  • Receptors, Lipoprotein / metabolism

Substances

  • Apolipoproteins B
  • Lipoproteins, HDL
  • Lipoproteins, LDL
  • RNA, Small Interfering
  • Receptors, Lipoprotein
  • Cholesterol