动物造模,灵长类杜氏肌萎缩症模型 z-bio 2021-10-19 694

　　Muscular dystrophy (DMD) is a recessive genetic disease of the X chromosome chain, caused by the loss of dystrophin function due to mutations in the dystrophin gene of muscle protein. The incidence of newborn boys is 1/30000. As we age, the disease continues to exacerbate muscle atrophy, eventually leading to death. Currently, there is no effective treatment for DMD.

　　Through the application of the latest CRISPR/Cas9 gene targeting technology and improved experimental methods, the Li Xiaojiang research team of the Institute of Genetic Development Biology of the Chinese Academy of Sciences and the Ji Weizhi team of the Yunnan Key Laboratory of Primate Biomedical Sciences have been established. Methods: A total of 11 newborn monkeys with mutations in the Duchenne dystrophin gene were obtained. Analysis of the muscle tissue of monkeys who died of muscular dystrophy showed that CRISPR/Cas9-mediated chimeric targeted mutations significantly reduced or eliminated the expression of dystrophin, which is similar to early muscle cell degeneration in DMD patients. It has been clearly shown that it has physiological characteristics.

　　After successfully applying CRISPR/Cas9 gene targeting technology to target specific genes in non-human primates, this study found that CRISPR/Cas9 gene targeting technology functionally knocked out the monkey genes of patients. This is our first time. Prove that we can simulate pathological changes. At the same time, the DMD monkey model can further reveal the important early pathological changes of DMD disease and provide an important animal model for early interventional treatment of the disease in the future.