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Article

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

16 Sept 2013

Abstract excerpt

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, inhibitors against factor IX (FIX) are difficult to eradicate due to anaphylactic reactions and nephrotic syndrome and...

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PMID: 24106230
DOI: 10.1002/emmm.201302859

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