Journal
NATURE
Volume 520, Issue 7546, Pages 186-U98Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nature14299
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Funding
- National Institute of General Medical Sciences [T32GM007753]
- Paul and Daisy Soros Fellowship
- US Department of Energy Computational Science Graduate Fellowship
- United States Public Health Service from the National Institutes of Health [RO1-GM34277, R01-CA133404]
- United States Public Health Service from the National Cancer Institute [PO1-CA42063]
- Cancer Center from the National Cancer Institute [P30-CA14051]
- National Institutes of Health through NIMH [5DP1-MH100706]
- National Institutes of Health through NIDDK [5R01DK097768-03]
- Waterman Award from the National Science Foundation
- Keck Foundation
- New York Stem Cell Foundation
- Damon Runyon Foundation
- Searle Scholars Foundation
- Merkin Foundation
- Vallee Foundation
- NIH core grant [5P30EY012196-17]
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The RNA-guided endonuclease Cas9 has emerged as a versatile genom-editing platform. However, the size of the commonly used Cas9 from Streptococcus pyogenes (SpCas9) limits its utility for basic research and therapeutic applications that use the highly versatileaderio-assolated virus (AAV) delivery vehicle. Here, we we characterize six smaller Cas9 ortholocgues and show that Cas9 from Staphylococcus aureus (SaCas9) can edit the genome with efficiencies similar to those of spcas9, while being more than 1 kilobase shorter. We packaged SaCas9 and its single guide RNA expression cassette into a single AAV vector and targeted the cholesterol regulatory gene Pcsk9 in the mouse liver. Within one week of injection we observed >40%. gene modification, accompanied by significant reductions in serum Pcsk9 and total cholesterol le; Is We further assess the genome -wide targeting specificity of SaCas9 and SpCas9 using BLESS, and demonstrate that SaCas9-mediated in vivo genome editing has the potential to be efficient and specific.
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