Aptamers as Therapeutics

Nat Rev Drug Discov. 2010 Jul;9(7):537-50. doi: 10.1038/nrd3141.

Abstract

Nucleic acid aptamers can be selected from pools of random-sequence oligonucleotides to bind a wide range of biomedically relevant proteins with affinities and specificities that are comparable to antibodies. Aptamers exhibit significant advantages relative to protein therapeutics in terms of size, synthetic accessibility and modification by medicinal chemistry. Despite these properties, aptamers have been slow to reach the marketplace, with only one aptamer-based drug receiving approval so far. A series of aptamers currently in development may change how nucleic acid therapeutics are perceived. It is likely that in the future, aptamers will increasingly find use in concert with other therapeutic molecules and modalities.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Aptamers, Nucleotide / chemistry
  • Aptamers, Nucleotide / therapeutic use*
  • Aptamers, Nucleotide / toxicity
  • DNA / chemistry
  • DNA / therapeutic use
  • Drug Discovery
  • Drug Evaluation, Preclinical
  • Genetic Techniques
  • Humans
  • Ligands
  • Nucleic Acid Conformation
  • RNA / chemistry
  • RNA / therapeutic use
  • RNA, Small Interfering / therapeutic use

Substances

  • Aptamers, Nucleotide
  • Ligands
  • RNA, Small Interfering
  • RNA
  • DNA