Using an improved CRISPR gene editing platform to study human development

  In a new study, researchers from the Sanger Institute of the Welcome Foundation and the University of Cambridge have constructed sOPTiKO, a more effective and controllable CRISPR genome editing platform. They explained how this free one-step system works on all cells and stages of the human body. This new method can help scientists in developmental biology, tissue regeneration and cancer research.

  has developed two complementary methods. sOPiTKO is a knockout system that can shut down genes by disrupting DNA. sOPTiKD is a knockdown system that can disrupt the effect of RNA silencing genes. Using these two methods, scientists can inductively switch off or silence genes at any stage of cell development, from stem cells to fully differentiated adult cells. This enables scientists from all over the world to quickly and accurately study the role of genes in the development of these cells to tissues such as the liver, skin, and heart, and discover how they promote health and disease. I can do it. The human body contains about 37 trillion cells, while the human genome contains only about 20,000 genes. Therefore, in order to produce all tissues and cell types in the body, different combinations of genes must be effective at different moments of organ or tissue development. Genes can be turned off at specific moments in cell development, allowing people to study their effects. The author of the paper, Professor Ludwig Barrier of the Cambridge Stem Cell Institute of the University of Cambridge Welcome Foundation Medical Research Committee, said: "When a cell grows from a stem cell to a fully differentiated adult cell, the genes in it show different Function. In the past, if you knock out a gene, you will only see how it affects you in the first step. No. You can make the gene play a role in cell development, and use sOPTiKO for gene in subsequent developmental steps Knock it out and accurately study its effect at that stage."

  SOPTiKO and sOPTIKD. This method allows scientists to silence the activity of multiple genes at once. Therefore, the entire relevant gene can be knocked down at once. Study the impact of family activities.

  Moreover, this freely available system makes experiments faster and cheaper. Due to its flexibility, sOPTiKO can be used in any tissue of the human body without having to build a new system every time. sOPiTKD greatly improves efficiency. It can be used to knock out multiple genes simultaneously. In the past, in order to suppress the activity of three genes, researchers knocked down one gene, cultivated a cell line, and then repeated the previous steps for the second gene, and finally the third gene. I have to repeat it. .. Now this can be done in just one step, reducing the 9-month process to one or two months. Co-author Dr. Alessandoberto said: "Compared with other CRISPR editing systems, the two main advantages of using OPTiKO/sOPTIKD are that it is truly inducible and can be used in almost all cell types. In the past, you could only study one gene in a specific Functions in tissues. Today, you can simultaneously knock out the same gene in multiple cell types with different functions.