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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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Bibliografiska uppgifter
Huvudupphovsmän: Markusic, David M, Hoffman, Brad E, Perrin, George Q, Nayak, Sushrusha, Wang, Xiaomei, LoDuca, Paul A, High, Katherine A, Herzog, Roland W
Materialtyp: Artigo
Språk:Inglês
Publicerad: Blackwell Publishing Ltd 2013
Ämnen:
Länkar: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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