Mouse model studies suggest that genome editing stops genetic diseases before conception

  Gene editing is the latest technology that can precisely modify the genome. Since its introduction, scientists have been committed to applying this technology to the research and treatment of human genetic diseases. Two previous studies published in Cell Research and PLOS ONE both demonstrated the use of genome editing technology to edit the genome of sperm stem cells and treat genetic diseases.

  Recently, scientists in the Department of Biochemistry at the University of Bath are expected to develop new technologies that can simplify biomedical research and prevent the development of genetic diseases before conception.

  Recently, related research results have been published in the sub-journal "Science Reports" of "Nature". In this study, scientists used "molecular scissors" to edit the DNA of mouse eggs and sperm during fertilization. The researchers used the Cas9 enzyme to cut the exact point in the genome so that they can accurately suppress specific genes. In this way, scientists can prepare a "knockout" mouse model to study the function of a specific gene in nearly a month, compared with six months using traditional techniques.

  This powerful tool is expected to accelerate biomedical research and reduce the number of animals used in basic medical experiments. The senior author of this article, Dr. Tony Perry of the Institute of Mammalian Molecular Development, explained: "This research makes me very excited. Previously, this technique has been proven in mature embryos, but before and after fertilization, Before the embryo develops, the sperm or egg genes are accurately edited."

  " The Cas9 enzyme works by cutting DNA at the exact location in the genome. Cells can repair the cut, but chew the frayed ends before reconnecting them."

  "This technology has many exciting potential uses. They can provide disease resistance to livestock, perhaps as a way to do so. By preventing serious genetic diseases close to pregnancy (such as suffering from fatal genetic diseases (such as cystic fiber) To avoid the risk of spreading the disease to the offspring. Researchers can also use this method to transplant some large animal organs into humans, thereby masking the immunity of the organs and avoiding rejection reaction.

  Dr. Perry added: For patients waiting for transplant surgeons and immune-matched organs, this is a dream: these artificial organs can be transplanted until a matching human organ appears.