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Trio Deep-sequencing does not Reveal Unexpected off-target and on-target Mutations in Cas9-edited Rhesus Monkeys
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Luo X, He Y, Zhang C, He X, Yan L, Li M, Hu T, Hu Y, Jiang J, Meng X, Ji W, Zhao X, Zheng P, Xu S, Su B |
Abstract: |
CRISPR-Cas9 is a widely-used genome editing tool, but its off-target effect and on-target complex mutations remain a concern, especially in view of future clinical applications. Non-human primates (NHPs) share close genetic and physiological similarities with humans, making them an ideal preclinical model for developing Cas9-based therapies. However, to our knowledge no comprehensive in vivo off-target and on-target assessment has been conducted in NHPs. Here, we perform whole genome trio sequencing of Cas9-treated rhesus monkeys. We only find a small number of de novo mutations that can be explained by expected spontaneous mutations, and no unexpected off-target mutations (OTMs) were detected. Furthermore, the long-read sequencing data does not detect large structural variants in the target region
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2019 |
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152435 |
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Nature Communications |
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https://www.nature.com/articles/s41467-019-13481-y |
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