Potent and systematic RNAi mediated silencing with single oligonucleotide compounds

RNA. 2011 Jun;17(6):1032-7. doi: 10.1261/rna.2399411. Epub 2011 Apr 14.

Abstract

RNA interference (RNAi) has been established as an important tool for functional genomics studies and has great promise as a therapeutic intervention for human diseases. In mammalian cells, RNAi is conventionally induced by 19-27-bp RNA duplexes generated by hybridization of two complementary oligonucleotide strands (oligos). Here we describe a novel class of RNAi molecules composed of a single 25-28-nucleotide (nt) oligo. The oligo has a 16-nt mRNA targeting region, followed by an additional 8-10 nt to enable self-dimerization into a partially complementary duplex. Analysis of numerous diverse structures demonstrates that molecules composed of two short helices separated by a loop can efficiently enter and activate the RNA-induced silencing complex (RISC). This finding enables the design of highly effective single-oligo compounds for any mRNA target.

MeSH terms

  • Cells, Cultured
  • Gene Silencing
  • HeLa Cells
  • Humans
  • Nucleic Acid Conformation
  • Oligonucleotides / chemistry*
  • RNA / chemistry
  • RNA / metabolism
  • RNA Interference*
  • RNA, Messenger / chemistry
  • RNA, Messenger / metabolism
  • RNA, Small Interfering / chemistry*
  • RNA, Small Interfering / metabolism
  • RNA-Induced Silencing Complex / chemistry
  • RNA-Induced Silencing Complex / metabolism*

Substances

  • Oligonucleotides
  • RNA, Messenger
  • RNA, Small Interfering
  • RNA-Induced Silencing Complex
  • RNA