Release date: 2017-06-29
A Japanese research team recently reported that they successfully treated hemophilia in experimental mice using gene editing technology. In the future, it is expected to develop a therapy that can cure hemophilia.
Hemophilia is a hemorrhagic disease caused by genetic abnormalities, which is characterized by the inability of the human body to produce coagulation factors or insufficient production of coagulation factors, resulting in prolonged clotting time and difficulty in stopping bleeding. In order to prevent bleeding, some patients have to take 1 to 3 times a week to inject coagulation factor preparations.
Researchers at institutions such as the Japan Autonomous Medical University recently published a paper in the British "Science Report" magazine, saying that researchers first developed genetically abnormal hemophilia experimental mice, and then used the most popular CRISPR/Cas9 gene editing technology. An enzyme and a normal gene capable of destroying abnormal genes were introduced into the liver site where the blood coagulation factor was produced, and the abnormal gene was successfully repaired, and the hemophilia of the experimental mouse was cured.
The researchers said that this is only the result of animal experiments, and I hope to develop a method to cure hemophilia on this basis. The researchers said that there are individual differences in genetic variation, and if this method can be used clinically in the future, targeted genetic repair may be required for different people.
Source: Xinhua News Agency
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