Reversal of siRNA-mediated gene silencing in vivo

Nat Biotechnol. 2018 Jul;36(6):509-511. doi: 10.1038/nbt.4136. Epub 2018 May 14.

Abstract

We report rapid, potent reversal of GalNAc-siRNA-mediated RNA interference (RNAi) activity in vivo with short, synthetic, high-affinity oligonucleotides complementary to the siRNA guide strand. We found that 9-mers with five locked nucleic acids (LNAs) have the highest potency across several targets. Our modular, sequence-specific approach, named REVERSIR, may enhance the therapeutic profile of any long-acting GalNAc-siRNA (short interfering RNA) conjugate by enabling control of RNAi pharmacology.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetylgalactosamine / genetics
  • Animals
  • Base Sequence
  • Biotechnology
  • Cells, Cultured
  • Female
  • Gene Silencing*
  • Hepatocytes / metabolism
  • Humans
  • Ligands
  • Mice
  • Mice, Inbred C57BL
  • Oligonucleotides / chemistry
  • Oligonucleotides / genetics
  • RNA Interference
  • RNA, Small Interfering / chemistry
  • RNA, Small Interfering / genetics*

Substances

  • Ligands
  • Oligonucleotides
  • RNA, Small Interfering
  • locked nucleic acid
  • Acetylgalactosamine