Gene therapy advances

Developments in the use of gene therapy to treat muscular dystrophy and haemophilia have been reported in the past week. A team of scientists at the Salk Institute in California announced in the American journal, Proceedings of the National Academy of Sciences, that a form of haemophilia in mice had been successfully cured by using a common virus to transport a clotting factor gene into liver cells. Meanwhile, researchers at the University of Pennsylvania Health System have developed a method of using gene therapy to treat muscle cells throughout a whole limb - a development which could have significant implications for the treatment of the wasting disease muscular dystrophy.

Professor Hansell Stedman, one of the authors of the report in the journal Nature Medicine, expressed his hope that the method could also have positive implications for the treatment of heart failure.