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Aptamers as therapeutics

Journal

NATURE REVIEWS DRUG DISCOVERY
Volume 9, Issue 7, Pages 537-550

Publisher

NATURE PUBLISHING GROUP
DOI: 10.1038/nrd3141

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Funding

  1. Welch Foundation [F-1654]
  2. National Institute of General Medical Sciences [R01GM077040]

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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.

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