Gene therapy with HSCs stabilizes factor VIII in hemophilia A

A single-center study tested gene therapy for severe hemophilia A using lentiviral vector-transduced autologous HSCs in five participants. Factor VIII activity correlated with vector copy numbers, with group 2 showing higher levels and no bleeding events during follow-up. The therapy demonstrated stable factor VIII expression, effective bleeding prevention, and manageable safety outcomes, including transient neutropenia and thrombocytopenia.

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