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

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Presentation on theme: "FIX It in One Go: Enhanced Factor IX Gene Therapy for Hemophilia B"— Presentation transcript:

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

2 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  , DOI: ( /j.cell ) Copyright © 2017 Elsevier Inc. Terms and Conditions

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