Progress in gene therapy to treat haemophilia has been impressive in the past few years. Gene therapy has been used to successfully treat haemophilia in dogs. A leading researcher from Philadelphia USA, Professor Katherine High, is examining the obstacles to successful gene therapy in human patients with haemophilia. She hopes that the problems may be overcome in the next five years to develop a successful gene transfer approach for sufferers of haemophilia.
"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 today.
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