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

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.

SUBMITTER: Scott DW

PROVIDER: S-EPMC3257353 | biostudies-literature | 2012 Feb

REPOSITORIES: biostudies-literature

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

Scott David W DW Lozier Jay N JN

British journal of haematology 20111107 3

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 ...[more]

PMID: 22055221

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