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Unlocking the potential of the human genome with RNA interference

Abstract

The discovery of RNA interference (RNAi) may well be one of the transforming events in biology in the past decade. RNAi can result in gene silencing or even in the expulsion of sequences from the genome. Harnessed as an experimental tool, RNAi has revolutionized approaches to decoding gene function. It also has the potential to be exploited therapeutically, and clinical trials to test this possibility are already being planned.

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Figure 1: RNA silencing pathways in different organisms.
Figure 2: Genome-wide screens using RNAi.
Figure 3: Proposed scheme for the treatment of HIV patients using lentiviral vectors to transduce anti-HIV shRNA genes into the patient's haematopoietic stem cells.

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Acknowledgements

We thank members of the RNAi community for making this an exciting field of research. G.J.H. thanks F. Rivas for comments on the manuscript and J. Duffy for help with the figures. J.J.R. thanks L. Scherer for suggestions on therapeutic applications of RNAi. G.J.H. is supported by an Innovator award from the US Army Breast Cancer Research Program and by grants from the NIH. J.J.R. is supported by grants from the NIH, NIAID and NHBLI.

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Hannon, G., Rossi, J. Unlocking the potential of the human genome with RNA interference. Nature 431, 371–378 (2004). https://doi.org/10.1038/nature02870

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