Gene therapy for hemophilia

Pediatric Blood & Cancer
Steven W Pipe

Abstract

Individuals with the inherited bleeding disorder hemophilia have achieved tremendous advances in clinical outcomes through widespread implementation of prophylactic replacement with safe and efficacious factor VIII and IX. However, despite this therapeutic approach, bleeds still occur, some with serious consequence, joint disease has not been eradicated, and patients have not yet been liberated from the need for regular intravenous infusions. The shift from protein replacement to gene replacement is offering great hope to achieve durable levels of plasma factor activity levels high enough to remove the risk for recurrent joint bleeding. For the first time, clinical trial results are showing promise for "curative" correction of the bleeding phenotype.

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Citations

Sep 27, 2018·Pediatric Blood & Cancer·Courtney D Thornburg
Oct 4, 2018·Expert Review of Hematology·Guy YoungSteven Pipe
Dec 6, 2018·FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology·Jerry K Y ChanCitra N Z Mattar
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Oct 14, 2019·European Journal of Haematology·Richard A MarlarDorothy M Adcock
Jun 8, 2018·Haemophilia : the Official Journal of the World Federation of Hemophilia·S W Pipe
May 30, 2020·Haemophilia : the Official Journal of the World Federation of Hemophilia·Annette E BowyerStefan Tiefenbacher
Jun 18, 2020·Research and Practice in Thrombosis and Haemostasis·Flora PeyvandiThierry VandenDriessche
Jun 6, 2019·JBJS Case Connector·Barbara MinkowitzJohn J Gregory

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