Activated factor IX Ρadua delivered via adeno-associated virus enhances therapeutic efficacy in hemophilia B

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1. 1An engineered AAVrh10 vector containing an optimized factor IX transgene improves therapeutic response during hemophilia b gene therapy2025
2. 2Increased gene therapy efficacy through the use of extended half-life clotting factorsClinical Trial · 2026 · 1 citations
3. 3Comparison of hemostatic dosing of factor IX wild-type and padua (R338L) in multiple hemophilia B mouse models2025
4. 4Pharmacokinetics, Biodistribution, Immunogenicity, and Model-Informed-Based PK/PD Model of a Next-Generation Advanced Novel Gene Therapy for Hemophilia2025 · 2 citations
5. 5Gene Therapy for Hemophilias: Opportunities and Challenges for the Clinical Laboratory2025 · 4 citations