On the left, F.IX structure derived from 1RFN PDB (Protein Data Bank) entry. Residue R338 (spacefilling) was mutated to A for gene transfer. On the right, F.IX clotting activity and blood loss in a tailclipping assay 4 weeks after delivery of AAV-wild type F.IX or AAV-R338A F.IX to hemophilia B mice. Activity is reported as percentage of normal mice. OD indicates absorbance of hemoglobin in the saline solution in which the tail was placed.

On the left, F.IX structure derived from 1RFN PDB (Protein Data Bank) entry. Residue R338 (spacefilling) was mutated to A for gene transfer. On the right, F.IX clotting activity and blood loss in a tailclipping assay 4 weeks after delivery of AAV-wild type F.IX or AAV-R338A F.IX to hemophilia B mice. Activity is reported as percentage of normal mice. OD indicates absorbance of hemoglobin in the saline solution in which the tail was placed.

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