Trio deep-sequencing does not reveal unexpected mutations in Cas9-edited monkeys

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.

doi:10.1101/339143

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bioRxiv · 10.1101/339143

Trio deep-sequencing does not reveal unexpected mutations in Cas9-edited monkeys

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.

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Abstract

CRISPR-Cas9 is a widely-used genome editing tool, but its off-target effect remains 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, no comprehensive in vivo off-target assessment has been conducted in NHPs. Here we performed whole genome trio sequencing of Cas9-treated monkeys. We found they only carried a small number of de novo mutations that can be explained by expected spontaneous mutations, and no unexpected mutations were detected.

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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.. 2018-06-05. Trio deep-sequencing does not reveal unexpected mutations in Cas9-edited monkeys. https://doi.org/10.1101/339143

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