Synthesis, characterization, and in vivo delivery of siRNA-squalene nanoparticles targeting fusion oncogene in papillary thyroid carcinoma

J Med Chem. 2011 Jun 23;54(12):4067-76. doi: 10.1021/jm2000272. Epub 2011 May 18.

Abstract

We report the conjugation of the natural lipid squalene (SQ) with a small interfering RNA (siRNA), against the junction oncogene RET/PTC1, usually found in papillary thyroid carcinoma (PTC). The acyclic isoprenoid chain of squalene has been covalently coupled with siRNA RET/PTC1 at the 3'-terminus of the sense strand via maleimide-sulfhydryl chemistry. Remarkably, the linkage of siRNA RET/PTC1 to squalene led to an amphiphilic molecule that self-organized in H(2)O as siRNA-SQ RET/PTC1 nanoparticles (NPs). The siRNA-SQ RET/PTC1 NPs, stable in H(2)O, were used for biological studies. In vitro, they did not show any cytotoxicity. Interestingly, in vivo, on a mice xenografted RET/PTC1 experimental model, RET/PTC1-SQ NPs were found to inhibit tumor growth and RET/PTC1 oncogene and oncoprotein expression after 2.5 mg/kg cumulative dose intravenous injections. In conclusion, these results showed that the "squalenoylation" offers a new noncationic plate-form for the siRNA delivery.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Carcinoma
  • Carcinoma, Papillary / genetics*
  • Carcinoma, Papillary / pathology
  • Carcinoma, Papillary / therapy
  • Cell Line, Tumor
  • Cell Survival
  • Drug Screening Assays, Antitumor
  • Humans
  • Hydrophobic and Hydrophilic Interactions
  • Mice
  • Mice, Nude
  • Nanoparticles*
  • Neoplasm Transplantation
  • Oligonucleotides / chemistry
  • Oncogene Proteins, Fusion / genetics*
  • Oncogene Proteins, Fusion / metabolism
  • Protein-Tyrosine Kinases / genetics*
  • Protein-Tyrosine Kinases / metabolism
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / chemistry
  • Squalene / chemistry*
  • Thyroid Cancer, Papillary
  • Thyroid Neoplasms / genetics*
  • Thyroid Neoplasms / pathology
  • Thyroid Neoplasms / therapy
  • Transplantation, Heterologous

Substances

  • Oligonucleotides
  • Oncogene Proteins, Fusion
  • RNA, Small Interfering
  • Squalene
  • Protein-Tyrosine Kinases
  • ret-PTC fusion oncoproteins, human