Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency
Cao, WJ; Dong, B; Horling, F; Firrman, JA; Lengler, J; Klugmann, M; de la Rosa, M; Wu, WM; Wang, QZ; Wei, HY; Moore, AR; Roberts, SA; Booth, CJ; Hoellriegl, W; Li, D; Konkle, B; Miao, C; Reipert, BM; Scheiflinger, F; Rottensteiner, H; Xiao, WD
Xiao, WD (corresponding author), Indiana Univ, Herman B Wells Ctr Pediat Res, Indianapolis, IN 46202 USA.; Rottensteiner, H (corresponding author), Baxalta Innovat GmbH, Donau City Str 7, A-1220 Vienna, Austria.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2020; 19 (): 486