Discovery of siRNA lipid nanoparticles to transfect suspension leukemia cells and provide in vivo delivery capability

Mol Ther. 2014 Feb;22(2):359-370. doi: 10.1038/mt.2013.210. Epub 2013 Sep 3.

Abstract

As a powerful research tool, siRNA's therapeutic and target validation utility with leukemia cells and long-term gene knockdown is severely restricted by the lack of omnipotent, safe, stable, and convenient delivery. Here, we detail our discovery of siRNA-containing lipid nanoparticles (LNPs) able to effectively transfect several leukemia and difficult-to-transfect adherent cell lines also providing in vivo delivery to mouse spleen and bone marrow tissues through tail-vein administration. We disclose a series of novel structurally related lipids accounting for the superior transfection ability, and reveal a correlation between expression of Caveolins and successful transfection. These LNPs, bearing low toxicity and long stability of >6 months, are ideal for continuous long-term dosing. Our discovery represents the first effective siRNA-containing LNPs for leukemia cells, which not only enables high-throughput siRNA screening with leukemia cells and difficult-to-transfect adherent cells but also paves the way for the development of therapeutic siRNA for leukemia treatment.

MeSH terms

  • Animals
  • Anions / chemistry
  • Cations / chemistry
  • Caveolins / genetics
  • Cell Line, Tumor
  • Disease Models, Animal
  • Gene Expression
  • Gene Transfer Techniques*
  • Humans
  • Leukemia / genetics
  • Lipids* / chemistry
  • Mice
  • Nanoparticles* / chemistry
  • Polymers / chemistry
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / chemistry
  • Transfection* / methods

Substances

  • Anions
  • Cations
  • Caveolins
  • Lipids
  • Polymers
  • RNA, Small Interfering