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Gene Therapy Corrects Factor VIII Levels in Hemophilia A

Posted on: 7 August 2017, source: Medscape
Patients with hemophilia A who received a gene for coagulation factor VIII (FVIII) packaged in a viral vector had sustained levels of FVIII during 1 year of observation, eliminating spontaneous bleeds[2]. Most of them did not need FVIII infusions even in the case of major trauma or surgery. In a late-breaking abstract session here at the International Society on Thrombosis and Hemostasis 2017 Congress, Dr John Pasi (Barts and the London School of Medicine and Dentistry, Queen Mary University of London, UK) said six out of the seven patients in one dose cohort have achieved normal factor VIII levels.