Article
In vitro rescue of FGA deletion by lentiviral transduction of an afibrinogenemic patient's hepatocytes.
Journal of thrombosis and haemostasis : JTH - 1 Nov 2014
Stroka D, Keogh A, Vu D, Fort A, Stoffel M H, Kühni-Boghenbor K, Furer C, Banz V, Demarmels Biasiutti F, Lämmle B, Candinas D, Neerman-Arbez M
Abstract excerpt
BACKGROUND: Congenital afibrinogenemia is a rare inherited autosomal recessive disorder in which a mutation in one of three genes coding for the fibrinogen polypeptide chains Aα, Bβ and γ results in the absence of a functional coagulation protein. A patient with congenital afibrinogenemia, resulting from an FGA homozygous gene deletion, underwent an orthotopic liver transplant that resulted in complete...
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