Directrice de recherche de l’unité Inserm 951 “Approches génétiques intégrées et nouvelles thérapies pour les maladies rares”
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Hemophilia is a serious hereditary disease that prevents the blood from clotting. This means that, in the event of a wound, bleeding doesn’t stop or is extremely difficult to stop.
There are two types of hemophilia:
– Hemophilia A, the more common form, is linked to a deficiency in clotting factor VIII
– Hemophilia B, occurring more rarely, is linked to a deficiency in clotting factor IX
Two clinical studies recently published in The New England Journal of Medicine show that it is now possible to cure both hemophilia A and hemophilia B through gene therapy. A one-hour transfusion of a rescue gene that codes for the faulty clotting factor was enough to cure patients suffering from the disease. This is a breakthrough in treating the disease, and it is also one step further for gene therapy, which is being used more and more often in clinical trials.
Multiple myeloma is a cancer of the bone marrow, with a life expectancy of less than 5 years post-diagnosis. While studying a neglected tropical disease, Buruli ulcer, researchers from....