Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B

Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate. AMT-060 combines an adeno-associated virus-5 (AAV5) vector with a liver-specific promoter driving expr...

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Udgivet i:Blood
Main Authors: Miesbach, Wolfgang, Meijer, Karina, Coppens, Michiel, Kampmann, Peter, Klamroth, Robert, Schutgens, Roger, Tangelder, Marco, Castaman, Giancarlo, Schwäble, Joachim, Bonig, Halvard, Seifried, Erhard, Cattaneo, Federica, Meyer, Christian, Leebeek, Frank W. G.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Hematology 2018
Fag:
Thrombosis and Hemostasis
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5833265/
https://ncbi.nlm.nih.gov/pubmed/29246900
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2017-09-804419
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