• Nature biotechnology · Sep 2015

    Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.

    • Ayal Hendel, Rasmus O Bak, Joseph T Clark, Andrew B Kennedy, Daniel E Ryan, Subhadeep Roy, Israel Steinfeld, Benjamin D Lunstad, Robert J Kaiser, Alec B Wilkens, Rosa Bacchetta, Anya Tsalenko, Douglas Dellinger, Laurakay Bruhn, and Matthew H Porteus.
    • Department of Pediatrics, Stanford University, Stanford, California, USA.
    • Nat. Biotechnol. 2015 Sep 1; 33 (9): 985-989.

    AbstractCRISPR-Cas-mediated genome editing relies on guide RNAs that direct site-specific DNA cleavage facilitated by the Cas endonuclease. Here we report that chemical alterations to synthesized single guide RNAs (sgRNAs) enhance genome editing efficiency in human primary T cells and CD34(+) hematopoietic stem and progenitor cells. Co-delivering chemically modified sgRNAs with Cas9 mRNA or protein is an efficient RNA- or ribonucleoprotein (RNP)-based delivery method for the CRISPR-Cas system, without the toxicity associated with DNA delivery. This approach is a simple and effective way to streamline the development of genome editing with the potential to accelerate a wide array of biotechnological and therapeutic applications of the CRISPR-Cas technology.

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