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Highly efficient and specific genome editing in human cells with paired CRISPR-Cas9 nickase ribonucleoproteins

Lamberth, J.; Daley, L.; Natarajan, P.; Khoruzhenko, S.; Becker, N.; Taglicht, D.; Davis, G. D.; Ji, Q.

2019-09-20 molecular biology
10.1101/766493 bioRxiv
Show abstract

CRISPR technology has opened up many diverse genome editing possibilities in human somatic cells, but has been limited in the therapeutic realm by both potential off-target effects and low genome modification efficiencies. Recent advancements to combat these limitations include delivering Cas9 nucleases directly to cells as highly purified ribonucleoproteins (RNPs) instead of the conventional plasmid DNA and RNA-based approaches. Here, we extend RNP-based delivery in cell culture to a less characterized CRISPR format which implements paired Cas9 nickases. The use of paired nickase Cas9 RNP system, combined with a GMP-compliant non-viral delivery technology, enables editing in human cells with high specificity and high efficiency, a development that opens up the technology for further exploration into a more therapeutic role.

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"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.