Liver gene therapy by lentiviral vectors reverses anti‐factor IX pre‐existing immunity in haemophili...
Liver gene therapy by lentiviral vectors reverses anti‐factor IX pre‐existing immunity in haemophilic mice
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London: Nature Publishing Group UK
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English
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London: Nature Publishing Group UK
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A major complication of factor replacement therapy for haemophilia is the development of anti‐factor neutralizing antibodies (inhibitors). Here we show that liver gene therapy by lentiviral vectors (LVs) expressing factor IX (FIX) strongly reduces pre‐existing anti‐FIX antibodies and eradicates FIX inhibitors in haemophilia B mice. Concomitantly, p...
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Liver gene therapy by lentiviral vectors reverses anti‐factor IX pre‐existing immunity in haemophilic mice
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TN_cdi_doaj_primary_oai_doaj_org_article_ebac0ce6ddd046b0865fecd8245a9d7e
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_ebac0ce6ddd046b0865fecd8245a9d7e
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1757-4676
E-ISSN
1757-4684
DOI
10.1002/emmm.201302857