The goal of gene therapy for hemophilia A is to safely transfer stable and durable expression of coagulation factor VIII (FVIII), which predictably improves the bleeding phenotype at the lowest possible vector dose. Updated data from a study examining the use of the adeno-associated viral vector (AAV) SPK-8011 were presented during the virtual congress of the International Society on Thrombosis and Haemostasis (ISTH) 2021.
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