The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

Also Consider

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

1. 1Factor IX variants improve gene therapy efficacy for hemophilia B2004 · 74 citations
2. 2Protein Replacement Therapy and Gene Transfer in Canine Models of Hemophilia A, Hemophilia B, von Willebrand Disease, and Factor VII Deficiency2009 · 73 citations
3. 3Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model2004 · 148 citations
4. 4AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B2003 · 757 citations
5. 5Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy2008 · 277 citations