Gene "magic shear" in non-human primates will not "bias" off target

Science and Technology Daily (Reporter Hanbin Zhao) CRISPR-Cas9 gene editing system has been widely used in biological and medical research, researchers are trying to use it for gene therapy of human diseases, however, it is not yet a comprehensive assessment of preclinical safety. Reporters from
Kunming Animal Research Institute learned that the recent cooperation of a number of research groups, found that this gene editing system in non-human primates will not lead to a significant off-target effect, and therefore have a higher safety. The findings are published in the latest issue of the journal Nature Communications.
previous studies have shown that crispr-Cas9 systems cause a large number of off-target mutations in gene editing in mice. In addition, studies of cell line have found large fragment structural variations in the target area of cas9 editing cells. However, these findings have been controversial due to problems with experimental design and data analysis, as well as the failure of rodents to characterize human disease in many ways.
macaques, as non-human primates that can be genetically operated recently in their relationship with human evolution, are highly similar to humans in terms of brain development and brain structure. Therefore, it is very important to explore the off-target effect of Cas9 gene editing in the monkey disease model for future clinical application.
Kunming Zoology Research Institute' Cebu Group, Zheng Ping Research Group, Primate Research Center and
Nutrition and Health Research Institute Xu Shuhua Task Force, using CRISPR-Cas9 system to build a microcephaly gene - MCPH1 knock-out of the macaque model. Through deep second-generation sequencing and analysis of multiple gene knock-out monkeys and their wild parents, it was found that Cas9 did not cause a large number of new mutations in the primate genome.
results show that these new mutations are not caused by Cas9. In addition, long fragment sequencing technology found that there is no structural variation of large fragments in the target area. These results show that the CRISPR-Cas9 gene editing system does not have obvious off-target effect in non-human primates and has a relatively high safety.