Study Summary
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor IX into patients with hemophilia B, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
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YUVA-GT-F901BIOLOGICAL
Lentiviral factor IX gene modified autologous hematopoietic and mesenchymal stem cells
Study Locations
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