Haemophilia gene therapy 'solution'
Dogs with the bleeding disorder haemophilia A have been successfully treated by gene therapy, according to US scientists.
Two of three dogs given the experimental treatment remain free of severe symptoms more than two years on, they write in Nature Communications.
Haemophilia A is a bleeding disorder which affects one in 10,000 men.
Gene therapy may be suitable for human treatment for bleeding disorders in 10 years, says the Haemophilia Society.
People with haemophilia A have an error in their genetic code, which means they cannot produce a protein called Factor VIII, which helps in blood-clotting.
Researchers in the US and France studied three dogs with a disorder similar to human haemophilia A.
They used a virus to carry the normal gene for factor VIII into platelets, the blood cells involved repairing damaged blood vessels.
More than two years after the gene therapy, two of the three dogs remained free of serious internal bleeding.
Lead researcher Dr David Wilcox of the Medical College of Wisconsin, Milwaukee, said the study could ultimately improve the quality of life of patients affected by haemophilia A.
Haemophilia patients usually suffer internal bleeding several times a year and have to have regular injections of synthetic clotting factor.
Two of three dogs given the experimental treatment remain free of severe symptoms more than two years on, they write in Nature Communications.
Haemophilia A is a bleeding disorder which affects one in 10,000 men.
Gene therapy may be suitable for human treatment for bleeding disorders in 10 years, says the Haemophilia Society.
People with haemophilia A have an error in their genetic code, which means they cannot produce a protein called Factor VIII, which helps in blood-clotting.
Researchers in the US and France studied three dogs with a disorder similar to human haemophilia A.
They used a virus to carry the normal gene for factor VIII into platelets, the blood cells involved repairing damaged blood vessels.
More than two years after the gene therapy, two of the three dogs remained free of serious internal bleeding.
Lead researcher Dr David Wilcox of the Medical College of Wisconsin, Milwaukee, said the study could ultimately improve the quality of life of patients affected by haemophilia A.
Haemophilia patients usually suffer internal bleeding several times a year and have to have regular injections of synthetic clotting factor.
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