血友病基因疗法,动物试验 z-bo 2020-11-05 355

　　Hemophilia is a disease caused by hereditary coagulation dysfunction. It is characterized by the body's inability to produce coagulation factors or insufficient production of coagulation factors, which leads 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 for producing this protein.

　　The research team of Kyoto University and Nara Prefectural Medical University used special transport molecules to implant the clotting factor gene into the liver of experimental mice with hemophilia. They found that the liver of the experimental mice began to produce clotting factors, and the effect lasted more than 300 days. . Moreover, the experimental mice can easily stop bleeding after bleeding, indicating that the blood clotting function can be restored.

　　At present, there is no fundamental 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 use this gene therapy to implant genes that control the production of clotting proteins into human induced pluripotent stem cells (iPS cells), differentiate into liver cells, and transplant them into the human body to treat patients with hemophilia. Induced pluripotent stem cells are stem cells that are cultivated by "reprogramming" mature cells and possess differentiation potential similar to embryonic stem cells.