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Article

Gene therapy for haemophilia B

25 Jun 2012

Abstract excerpt

AAV virus mediated transfer of factor IX to humans is safe and effective at three dose levels. Two subjects treated at highest dose level developed immune mediated transaminitis which resolved on a short course of Prednisolone. Beneficial effects in terms of continuous elevation of factor IX level above base line was seen in all subjects, continuing for over 18 months. Further study of this treatment method is...

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Identifiers

PMID: 22726076
DOI: 10.1111/j.1365-2516.2012.02823.x

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