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

Abstract

One important limitation for achieving therapeutic expression of human factor VIII (FVIII) in hemophilia A gene therapy is inefficient secretion of th......

Full Text Link


Links

期刊讨论 | 中国SCI论文 | 期刊主页 | 投稿经验 | 杂志官网 | 投稿链接 | 作者需知 | PMC链接 | Pubmed全文检索