CRISPR/Cpf1基因编辑,动物造模 z-bio 2022-01-31 814

　　Lai Liangxue's group from the Guangzhou Institute of Biomedical Sciences, Chinese Academy of Sciences published a new gene editing study in the international journal Cell and Molecular Life Sciences, "Engineering CRISPR/Cpf1withtRNA to promote genome editing capabilities in mammalian systems."

　　CRISPR/Cpf1 is a new type of RNA-guided gene editing tool. Compared with CRISPR/Cas9, which is widely used today, it is more specific, has a lower off-target rate, and Cpf1 can identify T-based enrichment intervals. Motif (PAM) has expanded the use of CRISPR gene editing systems. However, its targeting efficiency is lower than Cas9, which limits the practical application of Cpf1 in gene editing. At present, it has only been reported in human cell lines and mouse embryos, and has not been used to achieve large-scale effective gene editing. animal. In this study, the researchers were the first to modify the Cpf1 guide RNA (gRNA) using the endogenous splicing mechanism of mammalian transfer RNA (tRNA). This significantly improves the editing of the Cpf1 gene in cell lines and animal embryos. The research team applied a new and improved Cpf1 gene editing system and successfully obtained the first Cpf1 gene-modified rabbit and pig models.

　　Aging is one of the current research hotspots in the field of life sciences, and the current mammalian model is only a mouse model. Embryo injection edited the rabbit WRN gene, providing the world's first adult rabbit early aging model and providing a new animal model for aging research. In addition, through Cpf1-mediated somatic gene editing and nuclear transfer technology, the research team established two gene-edited pig models in a pig model of dilated cardiomyopathy caused by a point mutation in the PLN gene, namely the 14th spermine Acid and DMD. There is a pig model. The above two model pigs are Duchenne muscular dystrophy formed by gene knockout, which provide ideal large animal disease models for the development of new therapies for corresponding diseases (such as drug therapy, gene therapy and stem cell therapy).