Gene therapy gives hope to haemophiliacs

Dr. Chris Troedson, 35, received a cloned version of what's known as the Christmas disease gene, which expresses the blood-clotting agent Factor IX, deficient in some haemophiliacs because of a genetic defect inherited from their mother. The gene, which was encased in a harmless virus, known as adeno-associated virus (AAV), was injected via a catheter through his groin into the blood vessel which supplies the liver. The virus casing allowed the gene, cloned from an anonymous person's DNA, to infiltrate Dr Troedson's liver cells, telling them to make Factor IX.

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