动物实验,血友病,基因疗法 z-bio 2021-11-03 295

　　Hemophilia is a disease caused by hereditary coagulopathy. It is characterized in that the human body cannot produce coagulation factors or insufficient production of coagulation factors, which increases the clotting time and makes it difficult to stop 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 clotting factor genes into the livers of experimental mice with hemophilia, and the livers of the experimental mice began to produce clotting factors. We found that the effect can last 300 times. day. In addition, it has been demonstrated that experimental mice can easily stop bleeding and restore blood coagulation after bleeding.

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