Genome Editing

Toward better control of genome editing

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Science  29 May 2015:
Vol. 348, Issue 6238, pp. 986-987
DOI: 10.1126/science.348.6238.986-e

Thanks to the CRISPR-Cas system, scientists can now modify nearly any location in the genome. A stumbling block to therapeutic use, however, is that Cas9 (an endonuclease) cleaves DNA at areas of interest but often also at off-target sites. Scientists hope to overcome this by engineering versions of Cas9 with higher specificity for the target DNA. Zetsche et al. and Davis et al. explored regulating the activity of Cas9 through two different approaches, both of which relied on pharmacological activation of the enzyme. By restricting the amount of active Cas9, both strategies achieved increases in specificity. These and other specificity-enhancing approaches may give scientists the ability to better control genome modification.

Nat. Biotechnol. 33, 139 (2015); Nat. Chem. Biol. 11, 316 (2015).

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