Gene therapy provides safe, long-term relief for patients with severe hemophilia B
(MEMPHIS, Tenn. - November 19, 2014) Gene therapy developed at St. Jude Children's Research Hospital, University College London (UCL) and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported. The results appear in the November 20 edition of The New England Journal of Medicine.
In this study, participants received one of three doses of gene therapy that used a modified adeno-associated ...









