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

Spronck, EA; Liu, YP; Lubelski, J; Ehlert, E; Gielen, S; Montenegro-Miranda, P; de Haan, M; Nijmeijer, B; Ferreira, V; Petry, H; van Deventer, SJ

Liu, YP (reprint author), UniQure Biopharma BV, Paasheuvelweg 25A, NL-1105 BP Amsterdam, Netherlands.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2019; 15 (): 221

Abstract

Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and......

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