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RNA guides genome engineering

Nature Biotechnology volume 31, pages 208–209 (2013)Cite this article

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The Cas9 endonuclease is reprogrammed by RNAs for site-specific modification of eukaryotic and microbial genomes.

Last year, researchers interested in genome engineering took note of an intriguing new technology that relies on the bacterial endonuclease Cas9, which could be redirected to different target sites simply by modifying the sequence of a synthetic single guide RNA (sgRNA) complexed with the enzyme1. The approach, demonstrated on plasmid DNA in vitro1, has now been extended to genomic DNA in six studies published in Science2,3, Nature Biotechnology4,5,6 and eLife7. These reports prove the versatility of the Cas9-based system to modify the genomes of human and murine cell lines and of living organisms, including Escherichia coli and zebrafish, with efficiencies similar to those of established nucleases used for targeted genome engineering.

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Figure 1: Customizable molecular scissors.

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Authors and Affiliations

  1. Claudio Mussolino and Toni Cathomen are at the Laboratory of Cell and Gene Therapy, Center for Chronic Immunodeficiency, University Medical Center Freiburg, Freiburg, Germany.,

    Claudio Mussolino & Toni Cathomen

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  1. Claudio Mussolino
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  2. Toni Cathomen
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Correspondence to Toni Cathomen.

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The authors declare no competing financial interests.

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Mussolino, C., Cathomen, T. RNA guides genome engineering. Nat Biotechnol 31, 208–209 (2013). https://doi.org/10.1038/nbt.2527

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