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pro vyhledávání: '"Robert Z Gut"'
Autor:
Annette von Drygalski, Esteban Gomez, Adam Giermasz, Giancarlo Castaman, Nigel S Key, Susan S Lattimore, Frank W.G. Leebeek, Wolfgang A Miesbach, Michael Recht, Robert Z Gut, Ricardo Dolmetsch, Paul E Monahan, Sandra Le Quellec, Steven W Pipe
Publikováno v:
Blood advances. American Society of Hematology
Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene with a liver-specific promoter. Here we report 3-year outcomes from a Phas