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First received: June 27, 2023

Clinical Trial: Liver Biopsy Following Gene Therapy For Hemophilia

Study Status: NOT_YET_RECRUITING

Recruit Status: NOT_YET_RECRUITING

Condition: Hemophilia A

Study Type: OBSERVATIONAL


Official Title: Liver Biopsy Following Gene Therapy For Hemophilia

Brief Summary: This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have previously been treated with a factor VIII or factor IX gene-containing AAV-vector for liver-targeted gene transfer.Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion

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