Characterization of a genetically engineered mouse model of hemophilia A with complete deletion of the F8 gene.

ABSTRACT:

Unlabelled

ESSENTIALS: Anti-factor VIII (FVIII) inhibitory antibody formation is a severe complication in hemophilia A therapy. We genetically engineered and characterized a mouse model with complete deletion of the F8 coding region. F8(TKO) mice exhibit severe hemophilia, express no detectable F8 mRNA, and produce FVIII inhibitors. The defined background and lack of FVIII in F8(TKO) mice will aid in studying FVIII inhibitor formation.

Background

The most important complication in hemophilia A treatment is the development of inhibitory anti-Factor VIII (FVIII) antibodies in patients after FVIII therapy. Patients with severe hemophilia who express no endogenous FVIII (i.e. cross-reacting material, CRM) have the greatest incidence of inhibitor formation. However, current mouse

SUBMITTER: Chao BN 

PROVIDER: S-EPMC4755856 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature