One of these days I'm going to do a post on the holocaust that killed 95% of children with hemophilia by refusing to test blood for HIV. For now, this is a real first step to eliminating that dependence on human blood products....
https://goo.gl/85VDycA single infusion of a gene therapy for hemophilia A led to normalization of factor VIII levels that persisted for a year, British investigators reported here.
Six of seven patients treated with the highest dose of the engineered therapy had factor VIII levels >50 IU/dL, and the seventh patient had therapeutic levels of factor VIII at 1 year. The high-dose cohort averaged 16 bleeding episodes a year before treatment and one episode afterward.