Gene therapy in dogs has been shown to be effective against bleeding, both internal and external. This technique could be used by humans.
The fight against hemophilia is moving forward. Dogs with hemophilia have been successfully treated with gene therapy. A Franco-American team from the University of Wisconsin (United States) and Xavier Arnozan hospital in Pessac (Gironde) published the results on November 19 in the journal Nature Communications.
Hemophilia A is a disease manifested by spontaneous and / or prolonged bleeding. It affects one in 6,000 men and is caused by the wrong genetic code. Factor VIII (FVIII) does not activate during bleeding. It is precisely this protein that helps the blood to clot. Developed since the 1970s, gene therapy could activate inhibited genes in hemophiliacs. Researchers place great hopes in this. According to the Haemophilia Society, in Great Britain, it should be accessible to humans within ten years.
Virus injection
The Franco-American team treated three dogs carrying hemophilia A. The canine breed indeed reproduces the disease in the same way as humans. The researchers used a virus to transport FVIII into platelets, cells involved in repairing damaged blood vessels, through the spinal cord. The animals were then followed for more than two years. The course of the disease was examined. Two and a half after gene therapy, two of the three dogs were no longer showing symptoms of hemophilia. Serious internal bleeding no longer occurred in these subjects. No animal developed antibodies against FVIII, which could have limited the effectiveness of the treatment.
Better quality of life
Currently, there is no cure for this disease. However, some drugs allow you to live properly, especially by adding coagulants. According to principal investigator in the United States, Dr. David Wilcox, such a study could constitute a significant improvement in the lives of hemophiliac patients. Most of them suffer from internal bleeding several times a year. They must also inject synthetic coagulants.
With effective gene therapy, their quality of life would be greatly improved. “These results indicate that performing gene replacement therapy in patients’ spinal cord cells can lead to the production, storage and release of factor VIII from platelets to injury to prevent uncontrolled bleeding.” , several years after treatment, ”Dr Wilcox told BBC News.
There is still work to be done before we arrive at effective therapy. The researchers submitted a request for a clinical trial in the United States. It should take place within two years. This therapy could even be open to patients with liver problems, who do not have access to gene therapy via the liver. Already in 2011, six patients with hemophilia B had benefited from gene therapy, with injections through the liver. Four of them can now live without drug treatment.
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