Hemophilia is a serious inherited disease that makes it impossible for the blood to clot. When a person with hemophilia begins to bleed, the flow cannot stop or very hard. The consequences can be serious with severe bleeding in the event of an injury and sometimes the appearance of spontaneous bleeding, especially in the joints (called hemarthrosis).
There are two forms of hemophilia: hemophilia A is the most common. It affects one in every 5,000 births boys and is characterized by a deficiency of the protein that helps the blood to clot (clotting factor VIII).
Hemophilia cannot be cured and can only be controlled with treatments that involve injecting clotting agents. However, hope for treatment by gene therapy has just appeared, following a therapeutic trial carried out on dogs with hemophilia A (because dogs reproduce the disease in the same way as humans). A team of researchers from the University of Wisconsin (United States) and Xavier Arnozan Hospital in Pessac (Gironde) published the results in the journal Nature Communications.
The Franco-American team used a virus to transport the clotting factor in the platelets, via the spinal cord. The animals and the course of the disease were then followed for more than two years. At the end of this monitoring period, two of the three dogs no longer showed symptoms of hemophilia.
To continue their research, the scientists applied for a clinical trial in the United States. This could be achieved within the next two years.
