Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency

Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency

One important limitation for achieving therapeutic expression of human factor VIII (FVIII) in hemophilia A gene therapy is inefficient secretion of the FVIII protein. Substitution of five amino acids in the A1 domain of human FVIII with the corresponding porcine FVIII residues generated a secretion-...

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Bibliographic Details
Main Authors: Wenjing Cao, Biao Dong, Franziska Horling, Jenni A. Firrman, Johannes Lengler, Matthias Klugmann, Maurus de la Rosa, Wenman Wu, Qizhao Wang, Hongying Wei, Andrea R. Moore, Sean A. Roberts, Carmen J. Booth, Werner Hoellriegl, Dong Li, Barbara Konkle, Carol Miao, Birgit M. Reipert, Friedrich Scheiflinger, Hanspeter Rottensteiner, Weidong Xiao
Format: Article
Language:English
Published: Elsevier 2020-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
factor VIII
AAV
gene therapy
X5
secretion
vector
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120302175

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