动物造模,血友病 z-bio 2022-04-15 550

　　Hemophilia is a disease caused by hereditary coagulation dysfunction, which is characterized by the inability of the body to produce coagulation factors or insufficient production of coagulation factors, resulting in prolonged coagulation time and difficult to stop bleeding. Human clotting factor is primarily a protein produced by the liver, but the livers of people with hemophilia lack the normal gene for making this protein.

　　A research team from Kyoto University and Nara Prefectural University of Medicine used special transport molecules to implant coagulation factor genes into the livers of experimental mice with hemophilia. The results found that the livers of experimental mice began to produce coagulation factors, and the effect lasted for more than 300 days. . Moreover, the bleeding of the experimental mice was easily stopped, indicating that the coagulation function was restored.

　　At present, there is no fundamental treatment for hemophilia, and severe patients can only inject coagulation factor preparations every few days, which is expensive. In the future, the research team plans to use this gene therapy to implant genes that control the production of coagulation proteins into human induced pluripotent stem cells (iPS cells), differentiate into liver cells, and transplant them into humans to treat hemophilia patients. Induced pluripotent stem cells are stem cells created by "reprogramming" mature cells and have differentiation potential similar to embryonic stem cells.