Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec

Zhang, LN; Handyside, B; Murphy, R; Sihn, CR; Xie, L; Vitelli, C; Harmon, D; Siso, S; Liu, S; Bullens, S; Bunting, S; Fong, S

Fong, S (corresponding author), BioMarin Pharmaceut, Translat Biol, 105 Digital Dr, Novato, CA 94901 USA.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020; 17 (): 13

Abstract

AAV5-hFVIII-SQ (valoctocogene roxaparvovec) is an adeno-associated virus (AAV)-mediated gene therapy vector containing a B-domain-deleted human factor......

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