Journal
NATURE BIOTECHNOLOGY
Volume 33, Issue 9, Pages 985-U232Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nbt.3290
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Funding
- Myotonic Dystrophy Foundation
- Danish Council for Independent Research, Medical Sciences [DFF-1331-00735B, DFF-1333-00106B]
- Amon Carter Foundation
- Laurie Krauss Lacob Faculty Scholar Award in Pediatric Translational Research
- US National Institutes of Health [PN2EY018244, R01-AI097320]
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CRISPR-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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