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Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

Formation of pathogenic antibodies is a major problem in replacement therapies for inherited protein deficiencies. For example, antibodies to coagulation factors (‘inhibitors’) seriously complicate treatment of haemophilia. While immune tolerance induction (ITI) protocols have been developed, inhibi...

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Bibliografische gegevens
Hoofdauteurs: Markusic, David M, Hoffman, Brad E, Perrin, George Q, Nayak, Sushrusha, Wang, Xiaomei, LoDuca, Paul A, High, Katherine A, Herzog, Roland W
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Blackwell Publishing Ltd 2013
Onderwerpen:
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3840486/
https://ncbi.nlm.nih.gov/pubmed/24106230
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/emmm.201302859
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