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Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B

✍ Scribed by Harding, T C; Koprivnikar, K E; Tu, G H; Zayek, N; Lew, S; Subramanian, A; Sivakumaran, A; Frey, D; Ho, K; VanRoey, M J

Book ID: 110039437
Publisher: Nature Publishing Group
Year: 2004
Tongue: English
Weight: 288 KB
Volume: 11
Category: Article
ISSN: 0969-7128
DOI: 10.1038/sj.gt.3302142

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Background Several groups including our own have reported gene delivery to fetal organs by vector administration into the amniotic cavity. Based on these studies we hypothesised that the large surface of the fetal skin may be exploitable for high level production of systemically required gene produc