Phase 1 pilot study for hemophilia-A: AAV8 vector with prophylactic tacrolimus-glucocorticoid achieves therapeutic FVIII activity
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This prospective phase 1 pilot study evaluated the safety and efficacy of GS001, an adeno-associated virus serotype 8 (AAV8)-based vector encoding B-domain-deleted factor VIII with exploratory assessment of prophylactic immunosuppression. Twelve adult severe h... ...
