Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs

by Elisabeth A. Spronck, Ying Poi Liu, Jacek Lubelski, Erich Ehlert, Sander Gielen, Paula Montenegro-Miranda, Martin de Haan, Bart Nijmeijer, Valerie Ferreira, Harald Petry, Sander J. van Deventer
Published 2019-12-01