动物造模,血友病基因疗法 z-bio 2021-10-18 313

　　Hemophilia is a disease caused by hereditary clotting dysfunction. It is characterized by the human body's inability to produce clotting factors or lack of clotting factor production, leading to prolonged clotting time and difficult to stop bleeding. Human coagulation factor is mainly a protein produced by the liver, but the liver of hemophilia patients lacks the normal genes that produce this protein.

　　The research team of Kyoto University and Nara Prefectural Medical University used special transport molecules to implant the coagulation factor gene into the liver of experimental mice with hemophilia. The results showed that the ideal test liver began to produce coagulation factors, and the effect lasted more than 300 sky. In addition, the experimental mice can easily stop bleeding after bleeding, indicating that the blood coagulation function can be restored.

　　At present, there is no basic treatment for hemophilia. Critically ill patients can only inject coagulation factor preparations every few days, which is expensive. In the future, the research team plans to apply 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 the human body, and stop treatment for patients with hemophilia. Induced pluripotent stem cells are stem cells that have been cultivated by stopping "reprogramming" of mature cells, and have differentiation potential similar to embryonic stem cells.