FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B

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FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B David Lillicrap Cell Volume 171, Issue 7, Pages 1478-1480 (December 2017) DOI: 10.1016/j.cell.2017.11.049 Copyright © 2017 Elsevier Inc. Terms and Conditions

Figure 1 Gain-of-Function Factor IX Gene Therapy for Hemophilia B Peripheral vein administration of a novel AAV vector delivering the gain-of-function FIX Padua transgene results in persistent expression of FIX at levels sufficient to significantly influence bleeding rates and the requirement for FIX replacement therapy. Cell 2017 171, 1478-1480DOI: (10.1016/j.cell.2017.11.049) Copyright © 2017 Elsevier Inc. Terms and Conditions