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- W2037394865 endingPage "302" @default.
- W2037394865 startingPage "295" @default.
- W2037394865 abstract "Summary 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." @default.
- W2037394865 created "2016-06-24" @default.
- W2037394865 creator A5052704460 @default.
- W2037394865 creator A5058249371 @default.
- W2037394865 date "2011-11-07" @default.
- W2037394865 modified "2023-10-18" @default.
- W2037394865 title "Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation" @default.
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- W2037394865 doi "https://doi.org/10.1111/j.1365-2141.2011.08925.x" @default.
- W2037394865 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/3257353" @default.
- W2037394865 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/22055221" @default.
- W2037394865 hasPublicationYear "2011" @default.
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