A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice

A longstanding goal for the treatment of hemophilia B is the development of a gene transfer strategy that can maintain sustained production of clotting factor IX (F.IX) in the absence of an immune response. To this end, we have sought to use lentiviral vectors (LVs) as a means for systemic gene tran...

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Veröffentlicht in:	Blood 2007-12, Vol.110 (13), p.4144-4152
Hauptverfasser:	Brown, Brian D., Cantore, Alessio, Annoni, Andrea, Sergi, Lucia Sergi, Lombardo, Angelo, Della Valle, Patrizia, D'Angelo, Armando, Naldini, Luigi
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Antibodies Blood Cells - metabolism Factor IX - administration & dosage Factor IX - immunology Gene Transfer Techniques Genetic Therapy - methods Genetic Vectors - therapeutic use Hemophilia B - therapy Lentivirus - genetics Mice MicroRNAs - genetics MicroRNAs - pharmacology
Online-Zugang:	Volltext
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