Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

Nat Biotechnol. 2019 Jun;37(6):640-650. doi: 10.1038/s41587-019-0106-2. Epub 2019 Apr 29.

Abstract

The molecular mechanisms of toxicity of chemically modified phosphorothioate antisense oligonucleotides (PS-ASOs) are not fully understood. Here, we report that toxic gapmer PS-ASOs containing modifications such as constrained ethyl (cEt), locked nucleic acid (LNA) and 2'-O-methoxyethyl (2'-MOE) bind many cellular proteins with high avidity, altering their function, localization and stability. We show that RNase H1-dependent delocalization of paraspeckle proteins to nucleoli is an early event in PS-ASO toxicity, followed by nucleolar stress, p53 activation and apoptotic cell death. Introduction of a single 2'-O-methyl (2'-OMe) modification at gap position 2 reduced protein-binding, substantially decreasing hepatotoxicity and improving the therapeutic index with minimal impairment of antisense activity. We validated the ability of this modification to generally mitigate PS-ASO toxicity with more than 300 sequences. Our findings will guide the design of PS-ASOs with optimal therapeutic profiles.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Humans
  • Liver / drug effects
  • Oligonucleotides / chemistry*
  • Oligonucleotides / therapeutic use
  • Oligonucleotides, Antisense / chemistry*
  • Oligonucleotides, Antisense / therapeutic use
  • Phosphorothioate Oligonucleotides / chemistry*
  • Phosphorothioate Oligonucleotides / therapeutic use
  • Protein Binding / drug effects
  • Ribonuclease H / chemistry
  • Ribonuclease H / genetics
  • Therapeutic Index

Substances

  • Oligonucleotides
  • Oligonucleotides, Antisense
  • Phosphorothioate Oligonucleotides
  • locked nucleic acid
  • Ribonuclease H
  • ribonuclease HI