Rationally engineered Cas9 nucleases with improved specificity

IM Slaymaker, L Gao, B Zetsche, DA Scott, WX Yan… - Science, 2016 - science.org
IM Slaymaker, L Gao, B Zetsche, DA Scott, WX Yan, F Zhang
Science, 2016science.org
The RNA-guided endonuclease Cas9 is a versatile genome-editing tool with a broad range
of applications from therapeutics to functional annotation of genes. Cas9 creates double-
strand breaks (DSBs) at targeted genomic loci complementary to a short RNA guide.
However, Cas9 can cleave off-target sites that are not fully complementary to the guide,
which poses a major challenge for genome editing. Here, we use structure-guided protein
engineering to improve the specificity of Streptococcus pyogenes Cas9 (SpCas9). Using …
The RNA-guided endonuclease Cas9 is a versatile genome-editing tool with a broad range of applications from therapeutics to functional annotation of genes. Cas9 creates double-strand breaks (DSBs) at targeted genomic loci complementary to a short RNA guide. However, Cas9 can cleave off-target sites that are not fully complementary to the guide, which poses a major challenge for genome editing. Here, we use structure-guided protein engineering to improve the specificity of Streptococcus pyogenes Cas9 (SpCas9). Using targeted deep sequencing and unbiased whole-genome off-target analysis to assess Cas9-mediated DNA cleavage in human cells, we demonstrate that “enhanced specificity” SpCas9 (eSpCas9) variants reduce off-target effects and maintain robust on-target cleavage. Thus, eSpCas9 could be broadly useful for genome-editing applications requiring a high level of specificity.
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