Progress in gene therapy to treat haemophilia has been impressive inthe past few years. Gene therapy has been used to successfully treathaemophilia in dogs. A leading researcher from Philadelphia USA,Professor Katherine High, is examining the obstacles to successful genetherapy in human patients with haemophilia. She hopes that the problemsmay be overcome in the next five years to develop a successful genetransfer approach for sufferers of haemophilia.
"It has taken approximately 5 to 8 years to move from a cure forhaemophilia in mice to a cure in dogs. This has been achieved bymultiple gene transfer strategies. Clinical studies have identifiedwhich aspects of gene transfer therapy in dogs are directly applicablein humans and have identified potential problems, such as mode ofdelivery, which must be overcome before applying this approach inhumans," said High.
Professor High will review these exciting findings and thesteps to achieving a successful outcome in humans at the XXth Congressof the International Society on Thrombosis & Haemostasis in Sydneytoday.
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