Study bolsters ‘turbocharged’ protein as a promising tool in hemophilia gene therapy

12 marzo 2015

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. In addition, the ‘turbocharged’ clotting factor protein eliminated pre-existing antibodies that often weaken conventional treatments for people with hemophilia