The Medicinal Chemistry of Therapeutic Oligonucleotides

J Med Chem. 2016 Nov 10;59(21):9645-9667. doi: 10.1021/acs.jmedchem.6b00551. Epub 2016 Jul 29.

Abstract

Oligonucleotide-based therapeutics have made rapid progress in the clinic for treatment of a variety of disease indications. Unmodified oligonucleotides are polyanionic macromolecules with poor drug-like properties. Over the past two decades, medicinal chemists have identified a number of chemical modification and conjugation strategies which can improve the nuclease stability, RNA-binding affinity, and pharmacokinetic properties of oligonucleotides for therapeutic applications. In this perspective, we present a summary of the most commonly used nucleobase, sugar and backbone modification, and conjugation strategies used in oligonucleotide medicinal chemistry.

Publication types

  • Review

MeSH terms

  • Chemistry, Pharmaceutical
  • Humans
  • Models, Molecular
  • Molecular Conformation
  • Oligonucleotides / chemical synthesis
  • Oligonucleotides / chemistry
  • Oligonucleotides / therapeutic use*

Substances

  • Oligonucleotides