动物造模,血友病,动物试验,基因疗法 z-bio 2021-09-29 244

　　Hemophilia is a disease caused by hereditary coagulopathy, which is characterized by the body's inability to produce coagulation factors or insufficient production of coagulation factors, resulting in prolonged clotting time and reduced bleeding. Human coagulation factor is a protein mainly produced in the liver, but the liver of hemophilia patients lacks the normal genes that produce this protein.

　　The research team of Kyoto University and Nara Medical University used a special transport molecule to transplant the clotting factor gene into the liver of hemophilia laboratory mice. The liver of the laboratory mice produced a clotting factor. The effect lasted for 300 days. It also shows that experimental mice can easily stop bleeding and restore blood coagulation after bleeding.

　　At present, there is no cure for hemophilia. Critically ill patients only need to inject coagulation factor preparations every few days, which is expensive. In the future, the research team will use this gene therapy to transplant genes that control the production of coagulation proteins into human induced pluripotent stem cells (iPS cells), differentiate them into hepatocytes, and transplant them into the human body. I plan to treat patients. Hemophilia. Induced pluripotent stem cells are stem cells cultured by "reprogramming" mature cells and have the same differentiation potential as embryonic stem cells.