小鼠模型,基因组编辑 z-bo 2020-10-19 295

　　Gene editing is the latest technology that allows precise modification of the genome. Since its emergence, scientists have been working hard to apply this technology to the research and treatment of human genetic diseases. Two previous studies published in Cellesearch and PLOSONE showed that genome editing technology was used to edit the genome of sperm stem cells to treat genetic diseases.

　　Recently, scientists from the Department of Biology and Chemistry at the University of Bath in the UK have developed new technologies that are expected to simplify biomedical research before conception and prevent the development of genetic diseases.

　　Related research results were recently published in the sub-journal "Science Reports" of "Nature". In this study, scientists used "molecular scissors" to edit the DNA of mouse eggs or sperm during fertilization. The researchers used Cas9 enzymes to cut out the exact points in the genome so that they can precisely suppress specific genes. In this way, scientists can prepare a "knockout" mouse model to study the function of a specific gene, which takes nearly a month, while using traditional techniques requires six months.

　　This powerful tool is expected to accelerate biomedical research and reduce the number of animals used in basic medical experiments.

　　The lead author of this article and Dr. Tony Perry of the Institute of Mammalian Molecular Embroidery, explained: “We are very excited about this research. Previously, this technique has been proven in mature embryos, but we proved that before embryos are fertilized And before fertilization, we can do this. It can precisely edit the sperm and egg genes,"

　　" can work by cutting DNA at the exact location of the genome. Cells can repair this cut, but it can. It bites the frayed edge and then reconnects, destroying the function of the gene."

　　"The technology has many exciting potential uses to provide disease resistance to livestock. You can provide a method, or you can suffer from serious genetic diseases during pregnancy, for example, allowing lethal genetic diseases carriers (Such as cystic fibrosis) to give birth to healthy babies. To prevent and avoid the risk of disease transmission to offspring."

　　Researchers also hope that this method can be used to transplant some large animal organs into human organs. Avoid rejection problems through immunization.

　　Dr. Perry added: This is a dream for transplant surgeons and patients waiting for immune compatible organs. This means that one day these artificial organs can be transplanted until there is a matching human organ. More lives. "