Lipid-modified aminoglycoside derivatives for in vivo siRNA delivery

Adv Mater. 2013 Sep 6;25(33):4641-5. doi: 10.1002/adma.201301917. Epub 2013 Jun 27.

Abstract

Rationally designed siRNA delivery materials that are enabled by lipid-modified aminoglycosides are demonstrated. Leading materials identified are able to self-assemble with siRNA into well-defined nanoparticles and induce efficient gene knockdown both in vitro and in vivo. Histology studies and liver function tests reveal that no apparent toxicity is caused by these nanoparticles at doses over two orders of magnitude.

Keywords: aminoglycoside; drug delivery; gene knockdown; nanoparticle; siRNA.

Publication types

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

MeSH terms

  • Aminoglycosides / chemistry*
  • Animals
  • Gene Transfer Techniques*
  • HeLa Cells
  • Humans
  • Lipids / chemistry*
  • Mice
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism*

Substances

  • Aminoglycosides
  • Lipids
  • RNA, Small Interfering