Gene therapy for haemophilia: Prospects and challenges to prevent or reverse inhibitor formation

David W. Scott*, Jay N. Lozier

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

21 Scopus citations

Abstract

Monogenic hereditary diseases, such as haemophilia A and B, are ideal targets for gene therapeutic approaches. While these diseases can be treated with protein therapeutics, such as factor VIII (FVIII) or IX (FIX), the notion that permanent transfer of the genes encoding these factors can cure haemophilia is very attractive. An underlying problem with a gene therapy approach, however, is the patient's immune response to the therapeutic protein (as well as to the transmission vector), leading to the formation of inhibitory antibodies. Even more daunting is reversing an existing immune response in patients with pre-existing inhibitors. In this review, we will describe the laboratory and clinical progress, and the challenges met thus far, in achieving the goal of gene therapy efficacy, with a focus on the goal of tolerance induction.

Original languageEnglish
Pages (from-to)295-302
Number of pages8
JournalBritish Journal of Haematology
Volume156
Issue number3
DOIs
StatePublished - Feb 2012
Externally publishedYes

Keywords

  • Factor IX (FIX)
  • Factor VIII (FVIII)
  • Gene therapy
  • Haemophilia
  • Immune tolerance

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