血友病,动物实验 z-bo 2020-08-18 621

　　Hemophilia is a disease caused by hereditary coagulation dysfunction, which causes the body to fail to produce or produce insufficient coagulation factors, which leads to prolonged clotting time and difficulty in stopping bleeding. Human coagulation factor is a protein mainly produced by the liver, but the liver of hemophilia patients lacks the normal genes that produce 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. The liver of the experimental mice began to produce clotting factors, and I found that the effect continued. 300 days. In addition, it has been shown that experimental mice can easily stop bleeding and restore blood clotting function after bleeding.

　　There is currently no cure for hemophilia, and severely ill patients can only inject coagulation factors every few days, which is very expensive. In the future, the research team will use this gene therapy to transplant genes that regulate the production of clotting proteins into human induced pluripotent stem cells (iPS cells), differentiate into hepatocytes, and then transplant them into the human body. We plan to treat patients with hemophilia. Induced pluripotent stem cells are stem cells cultured with "reprogrammed" mature cells and have the same differentiation potential as embryonic stem cells.