In Vivo RNAi Efficacy of Palmitic Acid-Conjugated Dicer-Substrate siRNA in a Subcutaneous Tumor Mouse Model

Chem Biol Drug Des. 2016 Jun;87(6):811-23. doi: 10.1111/cbdd.12720. Epub 2016 Feb 15.

Abstract

Short interfering RNAs are used in RNA interference technology and are powerful tools for target gene silencing in a sequence-specific manner. In this study, we synthesized Dicer-substrate siRNAs consisting of 27-nt double-stranded RNAs conjugated with palmitic acid at the 5'-end of the sense strand and investigated their RNA interference efficacies in vitro and in vivo. The palmitic acid-conjugated 27-nt DsiRNAs (C16-Dsi27RNAs) were prepared by our simple synthesis strategy and achieved a good yield. C16-Dsi27RNAs showed enhanced in vitro RNA interference potency compared with not only non-modified Dsi27RNAs but also cholesterol-conjugated Dsi27RNAs against both an exogenous enhanced green fluorescent protein and the endogenous vascular endothelial growth factor gene in a human scirrhous-type gastric cancer cell line that stably expressed the enhanced green fluorescent protein gene (GCIY-eGFP). Additionally, C16-Dsi27RNAs had potent gene silencing activity against both enhanced green fluorescent protein and vascular endothelial growth factor as target genes in a subcutaneous tumor mouse model generated from GCIY-eGFP cells administered by intratumoral injection. These results suggest that the C16-Dsi27RNAs will be useful next-generation RNA interference molecules that can overcome the problems associated with RNA interference technology.

Keywords: Dicer-substrate siRNA; enhanced green fluorescent protein; in vivo RNAi activity; lipid-conjugated siRNAs; potent gene silencing; vascular endothelial growth factor.

Publication types

  • Editorial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line, Tumor
  • DEAD-box RNA Helicases* / genetics
  • DEAD-box RNA Helicases* / metabolism
  • Female
  • Gene Silencing*
  • Humans
  • Mice
  • Mice, Inbred BALB C
  • Mice, Nude
  • Neoplasm Proteins* / genetics
  • Neoplasm Proteins* / metabolism
  • Neoplasms, Experimental* / genetics
  • Neoplasms, Experimental* / metabolism
  • Neoplasms, Experimental* / therapy
  • Palmitic Acid / pharmacology*
  • RNA, Small Interfering* / genetics
  • RNA, Small Interfering* / pharmacology
  • Ribonuclease III* / genetics
  • Ribonuclease III* / metabolism

Substances

  • Neoplasm Proteins
  • RNA, Small Interfering
  • Palmitic Acid
  • DICER1 protein, human
  • Ribonuclease III
  • DEAD-box RNA Helicases