Inhibitors - cellular aspects and novel approaches for tolerance

D. W. Scott*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

17 Scopus citations


The immune response against therapeutic clotting factors VIII and IX (FVIII and FIX) is a major adverse event that can effectively thwart their effectiveness in correcting bleeding disorders. Thus, a significant number of haemophilia patients form antibodies, called inhibitors, which neutralize the procoagulant functions of therapeutic cofactors FVIII (haemophilia A) or FIX (haemophilia B). Understanding the cellular and molecular aspects of inhibitor formation is critical to designing tolerogenic therapies for clinical use. This review will focus on the basis of the immune response to FVIII, in particular, and will discuss emerging efforts to not only reduce immunogenicity but also to prevent and/or reverse inhibitor formation.

Original languageEnglish
Pages (from-to)80-86
Number of pages7
Issue numberS4
StatePublished - May 2014
Externally publishedYes


  • Factor VIII
  • Gene therapy
  • Haemophilia
  • Immunoglobulin fusions
  • Regulatory T cells


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