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Medicine

RNA as drug and antidote

Nature volume 419, pages 23–24 (2002)Cite this article

The biggest cause of serious illness and premature death in Western societies today is the blockage of blood vessels by abnormal blood clots1,2,3. On page 90 of this issue Rusconi and colleagues4 describe how they discovered a new drug that prevents blood clotting, and how they designed an antidote. The drug is an RNA 'aptamer', the sequence of which was found by iterative in vitro selection from a large combinatorial library of RNAs, screened for binding to a protein that promotes blood coagulation. Aptamers can be made of RNA or DNA, and they achieve their target-binding selectivity by virtue of the unique shapes they adopt through the formation of internal base pairs. Coagulation proteins are popular targets for both traditional and aptamer-based drugs, because they are available pure in soluble form, and their activity can be readily measured in assays of blood-clotting time or by their cleavage of small, colour-generating substrates5.

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Figure 1: How blood clots.

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  1. Faculty of Medicine, Imperial College of Science, Technology and Medicine, Haemostasis Research, MRC Clinical Sciences Centre, Hammersmith Hospital, Du Cane Road, London, W12 0NN, UK

    Edward Tuddenham

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  1. Edward Tuddenham
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Correspondence to Edward Tuddenham.

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Tuddenham, E. RNA as drug and antidote. Nature 419, 23–24 (2002). https://doi.org/10.1038/419023a

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