Gene therapy progress and prospects: RNA aptamers.

Journal Article (Journal Article;Review)

Aptamers are oligonucleotides evolved in vitro or in nature to bind target ligands with high affinity and specificity. They are emerging as powerful tools in the fields of therapeutics, drug development, target validation and diagnostics. Aptamers are attractive alternatives to antibody- and small-molecule-based therapeutics owing to their stability, low toxicity, low immunogenicity and improved safety. With the recent approval of the first aptamer drug Macugen by the US FDA, there is great impetus to develop therapeutic aptamers that can target a wide array of disease states. The recent demonstration that aptamer activity can be reversed by the administration of a simple antidote greatly enhances the potential value of aptamers as therapeutic agents.

Full Text

Duke Authors

Cited Authors

  • Que-Gewirth, NS; Sullenger, BA

Published Date

  • February 2007

Published In

Volume / Issue

  • 14 / 4

Start / End Page

  • 283 - 291

PubMed ID

  • 17279100

International Standard Serial Number (ISSN)

  • 0969-7128

Digital Object Identifier (DOI)

  • 10.1038/


  • eng

Conference Location

  • England