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Emerging clinical applications of RNA

Nature volume 418, pages 252–258 (2002)Cite this article

Abstract

RNA is a versatile biological macromolecule that is crucial in mobilizing and interpreting our genetic information. It is not surprising then that researchers have sought to exploit the inherent properties of RNAs so as to interfere with or repair dysfunctional nucleic acids or proteins and to stimulate the production of therapeutic gene products in a variety of pathological situations. The first generation of the resulting RNA therapeutics are now being evaluated in clinical trials, raising significant interest in this emerging area of medical research.

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Figure 1: Applications of trans-cleaving ribozymes for gene inhibition.
Figure 2: Trans-splicing-mediated repair of mutant transcripts.
Figure 3: RNA ligand-mediated inhibition of protein function.
Figure 4: Treatment of cancer patients with tumour RNA-transfected dendritic cells (DCs).

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  1. Department of Surgery, Duke University Medical Center, Durham, 27710, North Carolina, USA

    Bruce A. Sullenger & Eli Gilboa

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Correspondence to Bruce A. Sullenger.

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Sullenger, B., Gilboa, E. Emerging clinical applications of RNA. Nature 418, 252–258 (2002). https://doi.org/10.1038/418252a

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