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Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

Nature Reviews Genetics volume 10, pages 578–585 (2009)Cite this article

Abstract

New technologies are emerging that utilize artificial microRNA (miRNA) target sites to exploit or inhibit endogenous miRNA regulation. This approach has been used to improve cell-specific targeting for gene and stem cell therapy studies and for animal transgenics, and also to reduce the toxicity of oncolytic viruses and to attenuate viral vaccines. Artificial targets have also been used to sponge or decoy miRNAs as a way to study their functions. This article considers the benefits of this approach and design considerations for future studies.

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Figure 1: Using microRNA target sites to target transgene and viral expression.
Figure 2: Inhibiting endogenous microRNA function.

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Acknowledgements

We would like to thank A. Baccarini, R. Sachidanandam, A. Lombardo, M. De Palma, A. Cantore and B. Gentner for discussions. We apologize to those whose relevant work was not cited here. B.D.B. is supported by the National Institutes for Health (NIH) Diabetes Pathfinder Award (DP2DK083052-01) and L.N. is supported by Fondazione Telethon, the European Union (PERSIST, CONSERT, RIGHT) the NIH, and Associazione Italiana per la Ricerca Sal Cancro.

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  1. Brian D. Brown is at the Department of Genetics and Genomic Sciences, Mount Sinai School of Medicine, 1425 Madison Avenue, BOX 1498, New York, New York 10029, USA. brian.brown@mssm.edu,

    Brian D. Brown

  2. Luigi Naldini is at the San Raffaele Telethon Institute for Gene Therapy and Vita Salute San Raffaele University, San Raffaele Scientific Institute, Via Olgettina, 58, 20132 Milan, Italy. naldini.luigi@hsr.it,

    Luigi Naldini

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Brown, B., Naldini, L. Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet 10, 578–585 (2009). https://doi.org/10.1038/nrg2628

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