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Gene therapy progress and prospects: RNA aptamers

Gene Therapy volume 14, pages 283–291 (2007)Cite this article

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.

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Acknowledgements

We thank Juliana Layzer for reading the manuscript. Thanks also to DT Gewirth for help with the illustrations.

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

    N S Que-Gewirth & B A Sullenger

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  1. N S Que-Gewirth
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Correspondence to B A Sullenger.

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Conflict of interest

Bruce Sullenger is a scientific founder of Regado Biosciences Inc, a Biotechnology company focused upon the development of aptamer–antidote pairs.

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Que-Gewirth, N., Sullenger, B. Gene therapy progress and prospects: RNA aptamers. Gene Ther 14, 283–291 (2007). https://doi.org/10.1038/sj.gt.3302900

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