Review

Gene Therapy (2007) 14, 283–291. doi:10.1038/sj.gt.3302900

Gene therapy progress and prospects: RNA aptamers

N S Que-Gewirth1 and B A Sullenger1

1Department of Surgery, Duke University Medical Center, Durham, NC, USA

Correspondence: Dr BA Sullenger, Department of Surgery, Duke University Medical Center, Box 2601, Research Drive, Durham, NC 27710, USA. E-mail: b.sullenger@cgct.duke.edu

Received 30 May 2006; Revised 2 November 2006; Accepted 3 November 2006.

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Abstract

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.

Keywords:

aptamers, therapeutics, antidote, reversible drug

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