Study 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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Liver BiopsyPROCEDURE
Standard transjugular liver biopsy under moderate sedation
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| St. Jude Children's Research Hospital | Memphis | Tennessee | United States |