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- [45] SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B at the Low Vector Dose Achieved Sustained, Continuous Factor IX Activity Levels Adequate for Endogenous Prophylaxis Preventing Bleeding Episodes and Reducing the Risk of Immune Response MOLECULAR THERAPY, 2017, 25 (05) : 331 - 332
- [49] Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals with and without Adeno-Associated Virus Serotype 5 Neutralizing Antibodies (Titers <:700) with Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B BLOOD, 2022, 140 : 4904 - 4906