血友病,动物试验 z-bio 2021-07-07 716

　　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 long clotting time, bleeding, and it becomes difficult to stop. Human coagulation factor is a protein mainly produced in the liver, but the liver of hemophilia patients does not have the normal gene for producing this protein.

　　The research team at Kyoto University and Nara Medical University used a special transport molecule to transplant the clotting factor gene into the liver of experimental mice with hemophilia, and the liver of the experimental mice began to produce clotting factors. , I found that the effect lasts for 300 days. In addition, laboratory mice have been shown to easily stop bleeding after bleeding and restore blood clotting function.

　　Currently, there is no basic cure for hemophilia. Critically ill patients can only inject coagulation factor preparations every few days, which is expensive. In the future, this gene therapy will be used to transplant genes that control the production of coagulation proteins into human induced pluripotent stem cells (iPS cells) to differentiate into hepatocytes and transplant them into the human body to treat patients. What is going to be done. hemophilia. Induced pluripotent stem cells are stem cells cultured by "reprogramming" mature cells and have the same differentiation potential as embryonic stem cells.