GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

Background: GO-8 (ClinicalTrials.gov: NCT03001830) is a study of liver-directed adeno-associated virus (AAV) gene therapy for haemophilia A (HA) that uses a factor VIII (FVIII) variant containing a 17 amino-acid peptide comprising six N-linked glycosylation motifs from the human FVIII B-domain (AAV-...

Ausführliche Beschreibung

Bibliographische Detailangaben

Veröffentlicht in:	Blood 2023-11, Vol.142 (Supplement 1), p.3624-3624
Hauptverfasser:	Chowdary, Pratima, Reiss, Ulrike M., Tuddenham, Edward G. D., Batty, Paul, McIntosh, Jenny H., Radulescu, Vlad Calin, Chang, Eugenia, Laffan, Michael A., Riddell, Anne, Calvert, Joanna C. M., Pie, Arnulfo, Peralta, Rita, Dissanayake, Upuli Pabakumari, Kamel, Kirollos, Sreedhar, Bharath Ram, Zhou, Junfang, Kang, Guolian, Olufadi, Yunus, Meagher, Michael M, Morton, Christopher L, Evans, Alison J., Davidoff, Andrew M., Nathwani, Amit C.
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!