Impressive progress has been made in the treatment of haemophilia using gene therapy.

Professor Katherine High, a leading researcher from Philadelphia USA, is examining the obstacles to successful gene therapy in human patients with haemophilia.

Gene therapy has already been used to successfully treat haemophilia in mice and dogs.

“It has taken approximately 5 - 8 years to move from a cure for haemophilia in mice to a cure in dogs. This has been achieved by multiple gene transfer strategies. Clinical studies have identified which aspects of gene transfer therapy in dogs are directly applicable in humans and have identified potential problems, such as mode of delivery, which must be overcome before applying this approach in humans,” said High.

Professor High will review these exciting findings and the steps to achieving a successful outcome in humans at the XXth Congress of the International Society on Thrombosis & Haemostasis [] in Sydney this week.

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