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Tapping the RNA world for therapeutics

Abstract

A recent revolution in RNA biology has led to the identification of new RNA classes with unanticipated functions, new types of RNA modifications, an unexpected multiplicity of alternative transcripts and widespread transcription of extragenic regions. This development in basic RNA biology has spawned a corresponding revolution in RNA-based strategies to generate new types of therapeutics. Here, I review RNA-based drug design and discuss barriers to broader applications and possible ways to overcome them. Because they target nucleic acids rather than proteins, RNA-based drugs promise to greatly extend the domain of ‘druggable’ targets beyond what can be achieved with small molecules and biologics.

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Fig. 1: Antisense mechanisms of RNA-based drugs.

Debbie Maizels/Springer Nature

Fig. 2: Additional RNA-based drug mechanisms.

Debbie Maizels/Springer Nature

Fig. 3: RNA-based drug delivery strategies.

Debbie Maizels/Springer Nature

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Acknowledgements

This work was supported by NIH CA13944.

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Correspondence to Judy Lieberman.

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J.L. is on the Scientific Advisory Board of Alnylam Pharmaceuticals.

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Lieberman, J. Tapping the RNA world for therapeutics. Nat Struct Mol Biol 25, 357–364 (2018). https://doi.org/10.1038/s41594-018-0054-4

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