"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.