Gene therapy for haemophilia B

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...

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Veröffentlicht in:	Haemophilia : the official journal of the World Federation of Hemophilia 2012-07, Vol.18 (s4), p.13-17
1. Verfasser:	TUDDENHAM, E.
Format:	Artikel
Sprache:	eng
Schlagworte:	AAV Adenoviridae - genetics clinical trial Dependovirus Factor IX - administration & dosage Factor IX - genetics gene therapy Gene Transfer Techniques Genetic Therapy - methods Genetic Vectors haemophilia B Hemophilia B - genetics Hemophilia B - therapy Humans
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Beschreibung
Zusammenfassung:	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 warranted.
ISSN:	1351-8216 1365-2516
DOI:	10.1111/j.1365-2516.2012.02823.x