CRISPR/Cas9 z-bo 2020-10-20 289

　　In recent years, genome editing tools have been widely used, and clustered, regularly spaced short-interval palindrome repeat (CRISPR) technology has caused a surge in genome editing. CRISPR is a natural sequence in bacterial DNA that can cooperate with CRISPR-associated (Cas) nuclease to induce RNA and protect the bacterial genome from specific target sequences detected in invasive phage.

　　Recently, the original theory of using the high sequence specificity of this system to treat genetic diseases in animal models and prepare disease-free stem cells from diseased patients has been studied. CRISPR can also be used to prepare mouse models with specific genetic mutations. Compared with existing methods, this allows you to reach your research goals in a shorter time. According to reports, in early 2014, the CRISPR-Cas system was successfully used as a tool for efficient and highly specific genome-wide screening in human cells. Therefore, it opens up unlimited possibilities for finding genetic functions related to human health and disease.

　　For example, on February 5th, researchers from the Institute of Radiation and Radiology of the Academy of Military Medical Sciences published a new study in the journal Gene Therapy in the journal Nature, studying the encoding of the targeted HBV antigen (HBsAg). The impact of the CRISPR/CRISPR-related Cas9 system on cell culture systems and live animal areas. The results show that CRISPR/Cas9 can inhibit HBV replication and expression in vivo and in vitro, which may be a new treatment strategy for HBV infection. On March 12, researchers from the Salk Institute of Biology in the United States discovered for the first time a way to convert X-link SCID patients’ cells into a stem cell-like state, repair gene mutations and promote cell repair in the laboratory. I find. The successful production of K cells provides hope for the treatment of this rare disease. Extended reading: Nature: My scientists use CRISPR to destroy the hepatitis B virus; genome editing brings hope to rare diseases.

　　On April 9, the international journal "Human Molecular Genetics" of the Yunnan Key Institute of Primate Biomedicine, Institute of Genetics and Developmental Biology, Chinese Academy of Sciences, Kunming University of Science and Technology, Emory University School of Medicine. 》 Published a research paper entitled "Using CRISPR/Cas9 to Disrupt the Function of Rhesus Monkey Distrofingen". In this study, the CRISPR/Cas9 system was used to functionally disrupt the macaque dystrophin gene.

　　The corresponding authors of this article are Ji Weizhi, director of the Yunnan Institute of Primate Biomedical Sciences, and Dr. Li Xiaojiang, a researcher at the Institute of Genetic Development Biology, Chinese Academy of Sciences. Dr. Ji Weizhi used to be the deputy director, director and deputy director of the Kunming Research Office of the Chinese Academy of Sciences. I am the director of the Institute of Reproductive Biology. Biology/reproductive developmental biology research. In 2013, the Key Institute of Primate Biomedicine of Yunnan Province, led by the China Branch of Yunnan Province, used the world's latest genome editing technologies CRISPR/Cas9 and TALEN to carry out targeted genetic modification of two mackerel and cynomolgus monkeys. In November, we obtained the world’s first batch of two monkeys with CRISPR/Cas9 gene target modification and TALENs gene target modification, and published our research results on Cell and CellStemCell, which aroused the worldwide scientific community and media Wide attention. I'm.

　　related information He is engaged in the study of hereditary neurodegenerative diseases, intracellular transport and the pathological mechanism of early nervous system development, and his findings have been published many times. Applicable to PNAS, CellRes, HumMolGenet, Neuron, JCB, Brain, JBC and other internationally renowned journals. CRISPR/Cas9 has been used to genetically modify the genomes of various species including primates. Unfortunately, this new technology produced mosaic mutations. Whether such mutations functionally disrupt target genes or cause pathological signs in human diseases remains to be seen. These problems need to be solved when using CRISPR/Cas9 to create large animal models of human diseases.

　　In this study, researchers used CRISPR/Cas9 to target the dystrophin gene in rhesus monkeys to produce Duchenne muscular dystrophy (DMD, a recessive muscular dystrophy linked to X). mutation. The detection of relative targeting rate showed that CRISPR/Cas9 targeting can cause chimeric mutations in up to 87% of the dystrophin gene in the muscles of algae monkeys.

　　In addition, CRISPR/Cas9 can induce mutations in male and female rhesus monkeys, and find dystrophin and muscle degeneration in early DMD. These findings indicate that CRISPR/Cas9 can effectively prepare monkey models of human diseases regardless of the genetic model. Neonatal Cas9 targets degenerated muscle cells in red lizards, indicating that early intervention and treatment of the disease can effectively reduce myopathy.