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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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Autors principals: Markusic, David M, Hoffman, Brad E, Perrin, George Q, Nayak, Sushrusha, Wang, Xiaomei, LoDuca, Paul A, High, Katherine A, Herzog, Roland W
Format: Artigo
Idioma:Inglês
Publicat: Blackwell Publishing Ltd 2013
Matèries:
Accés en línia: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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