The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Coagulation Therapy in Hemophilia A and its Relation to Factor VIII Inhibitor in Northeast of Iran

Detection of Factor VIII Inhibitors in Hemophilia A Patients

Background: Factor VIII administration to hemophilia A patients results in an immune response (inhibitor formation) which significantly complicates the therapy. The present study was performed to determine the prevalence of inhibitor development in hemophilia A patients receiving recombinant factor VIII therapy. Materials and Methods: This was an observational descriptive study. Clotting fac...

Anti-CD3 antibodies modulate anti-factor VIII immune responses in hemophilia A mice after factor VIII plasmid-mediated gene therapy.

One major obstacle in gene therapy is the generation of immune responses directed against transgene product. Five consecutive anti-CD3 treatments concomitant with factor VIII (FVIII) plasmid injection prevented the formation of inhibitory antibodies against FVIII and achieved persistent, therapeutic levels of FVIII gene expression in treated hemophilia A mice. Repeated plasmid gene transfer is ...

coagulation therapy in hemophilia a and its relation to factor viii inhibitor in northeast of iran

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Hemophilia A gene therapy via intraosseous delivery of factor VIII-lentiviral vectors

Current treatment of hemophilia A (HemA) patients with repeated infusions of factor VIII (FVIII; abbreviated as F8 in constructs) is costly, inconvenient, and incompletely effective. In addition, approximately 25 % of treated patients develop anti-factor VIII immune responses. Gene therapy that can achieve long-term phenotypic correction without the complication of anti-factor VIII antibody for...