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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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Dettagli Bibliografici
Autori principali: Markusic, David M, Hoffman, Brad E, Perrin, George Q, Nayak, Sushrusha, Wang, Xiaomei, LoDuca, Paul A, High, Katherine A, Herzog, Roland W
Natura: Artigo
Lingua:Inglês
Pubblicazione: Blackwell Publishing Ltd 2013
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Accesso online: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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