MicroRNA-like off-target transcript regulation by siRNAs is species specific

RNA. 2009 Feb;15(2):308-15. doi: 10.1261/rna.1326809.

Abstract

siRNAs mediate sequence-specific gene silencing in cultured mammalian cells but also silence unintended transcripts. Many siRNA off-target transcripts match the guide-strand "seed region," similar to the way microRNAs match their target sites. The extent to which this seed-matched, microRNA-like, off-target silencing affects the specificity of therapeutic siRNAs in vivo is currently unknown. Here, we compare microRNA-like off-target regulations in mouse liver in vivo with those seen in cell culture for a series of therapeutic candidate siRNAs targeting Apolipoprotein B (APOB). Each siRNA triggered regulation of consistent microRNA-like off-target transcripts in mouse livers and in cultured mouse liver tumor cells. In contrast, there was only random overlap between microRNA-like off-target transcripts from cultured human and mouse liver tumor cells. Therefore, siRNA therapeutics may trigger microRNA-like silencing of many unintended targets in vivo, and the potential toxicities caused by these off-target gene regulations cannot be accurately assessed in rodent models.

Publication types

  • Comparative Study

MeSH terms

  • 3' Untranslated Regions / genetics*
  • 3' Untranslated Regions / metabolism
  • Animals
  • Apolipoproteins B / genetics*
  • Cell Line, Tumor
  • Cells, Cultured
  • Gene Expression Profiling
  • Gene Silencing*
  • Humans
  • Mice
  • MicroRNAs / genetics
  • MicroRNAs / metabolism*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism*
  • Selection, Genetic
  • Species Specificity
  • Transcription, Genetic

Substances

  • 3' Untranslated Regions
  • Apolipoproteins B
  • MicroRNAs
  • RNA, Small Interfering