AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

An AAV5 vector with a functional factor VIII gene was tested in men with hemophilia A. Men receiving the high dose had a factor VIII level above 5 IU per deciliter at 1 year; most had a normal level. Toxic effects were minimal, and bleeding episodes nearly eliminated.

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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2017-12, Vol.377 (26), p.2519-2530
Hauptverfasser:	Rangarajan, Savita, Walsh, Liron, Lester, Will, Perry, David, Madan, Bella, Laffan, Michael, Yu, Hua, Vettermann, Christian, Pierce, Glenn F, Wong, Wing Y, Pasi, K. John
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Alanine Alanine transaminase Antibodies, Viral - blood Bleeding Coagulation factors Dependovirus - immunology DNA, Viral Factor IX deficiency Factor VIII - administration & dosage Factor VIII - genetics Factor VIII - metabolism Gene therapy Genetic Therapy - adverse effects Genetic Therapy - methods Genetic Vectors Genomes Hemophilia Hemophilia A - genetics Hemophilia A - immunology Hemophilia A - metabolism Hemophilia A - therapy Hemorrhage - prevention & control Hemostasis Humans Infusions, Intravenous Intravenous administration Male Pharmaceuticals Quality of life Virus Shedding Young Adult
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Beschreibung
Zusammenfassung:	An AAV5 vector with a functional factor VIII gene was tested in men with hemophilia A. Men receiving the high dose had a factor VIII level above 5 IU per deciliter at 1 year; most had a normal level. Toxic effects were minimal, and bleeding episodes nearly eliminated.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1708483