CRISPR/Cas9,基因组编辑 z-bo 2020-09-18 298

　　Recently, genome editing tools have been widely used, and due to clustering, regularly spaced short-interval palindrome repeat (CRISPR) technology, genome editing has soared. 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, there is a unique evidence theory study that uses the high sequence specificity of the system to treat genetic diseases in animal models and prepare disease-free stem cells from affected patients. 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, and achieved target specificity in 2014. Great progress has been made in improvement. Therefore, it opens up unlimited possibilities for finding genetic functions related to human health and disease.

　　Researchers from the Institute of Radiology and the School of Radiology of the National Academy of Medical Sciences studied the coding of the targeted HBV antigen (HBsAg) in the "GeneTherapy" sub-journal of the journal Nature. Publish research. The impact of the CRISPR/CRISPR-related Cas9 system on cell culture systems and live animal areas. The results indicate that CRISPR/Cas9 may inhibit HBV replication and expression in vivo and in vitro, and may be a new treatment strategy for HBV infection. On March 12th, researchers from the Salk Institute of Biology in the United States showed how for the first time how to convert X-linked SCID patients’ cells into a stem cell-like state, repair gene mutations and promote cell repair in the laboratory. We found that the successful production of NK 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

　　Yunnan Institute of Primate Biomedicine, researchers from the Institute of Genetics and Developmental Biology of the Chinese Academy of Sciences, Kunming University of Science and Technology and Emory University School of Medicine have published in the international journal Human Molecular Genetics. 》 Published a research paper entitled "DistrophingeninhesusMonkeyUsingCRISPR/Cas9 Functional Disruption". In this study, the CRISPR/Cas9 system was used to functionally disrupt the monkey dystrophin gene. The corresponding authors of this document are Ji Weizhi, director of the Yunnan Provincial Key Institute of Primate Biomedicine, and Dr. Li Xiaojiang, a researcher at the Institute of Genetics and Developmental Biology, Chinese Academy of Sciences. Dr. Ji Weizhi used to be deputy director, director, deputy director, party committee member, committee member of Kunming Institute of Chinese Academy of Sciences, researcher of Kunming Institute of Chinese Academy of Sciences, Yunnan primate. He used to be the director of the Institute of Animal Breeding Biology. Biology/reproduction and developmental biology research. In 2013, he led the Yunnan Institute of Primate Biomedical Sciences, Ministry of Science and Technology of China, using the world's latest genome editing technologies CRISPR/Cas9 and TALEN to target mackerel and cynomolgus monkeys. Realize genetic recombination. In November, we obtained the world's first batch of two monkeys with CRISPR/Cas9 gene targeted modification and TALENs gene targeted modification. The research results were published on Cell and CellStemCell, and received extensive attention from the world's scientific community and media. Dr. Xiaojiang Li received his PhD from Oregon Health Science University in 1991. He is a doctoral researcher at Johns Hopkins University in the United States. He is engaged in the study of genetic neurodegenerative diseases, intracellular transport and the pathological mechanism of early nervous system development. His research results have been published many times. Applicable to PNAS, CellRes, HumMolGenet, Neuron, JCB, Brain, JBC and other internationally renowned journals. CRISPR/Cas9 is used for genetic recombination of the genomes of various species, including primates. Unfortunately, this new technology produced mosaic mutations. Whether this mutation can functionally destroy target genes or cause pathological signs seen in human diseases remains to be seen. When using CRISPR/Cas9 to prepare large animal models of human diseases, these problems need to be addressed.

　　In this study, the researchers used CRISPR/Cas9 to target the dystrophin gene in rhesus monkeys and caused Duchenne muscular dystrophy (DMD, X-linked recessive muscular dystrophy). The relative targeting rate test showed that CRISPR/Cas9 targeting can cause up to 87% of the dystrophin gene in the algae monkey muscle to have chimeric mutations. In addition, CRISPR/Cas9 can induce mutations in male and female monkeys. Dystrophin and muscle degeneration were found to be significantly reduced in early DMD. These findings indicate that no matter what the genetic model is, CRISPR/Cas9 can effectively prepare monkey models of human diseases. Neonatal Cas9 targets the degenerated muscle cells in red-tailed monkeys, which indicates that early intervention and treatment of the disease can effectively alleviate myopathy.