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Clustered, regularly interspaced short palindromic repeats (CRISPR) interference system for gene regulation

The CRISPR interference platform for modulating gene expression could be used to modify DNA in diverse biological systems. CRISPR systems use a small guide RNA (sgRNA), which pairs with a target DNA sequence and the CRISPR-associated protein (Cas9) to excise target DNA. In Escherichia coli cells, expression of a modified Cas9 without endonuclease activity plus sgRNA against a target gene led to reversible gene knockout. The same system also knocked down genes in human embryonic kidney cells. Next steps include testing and optimizing the CRISPR interference platform in patient-derived cells.

SciBX 6(10); doi:10.1038/scibx.2013.249
Published online March 14, 2013

Patent application filed; available for licensing

Qi, L.S. et al. Cell; published online Feb. 28, 2013;
Contact: Lei S. Qi, University of California, San Francisco, Calif.