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Scientific article
English

In vitro rescue of FGA deletion by lentiviral transduction of afibrinogenemic patient's hepatocytes

Published inJournal of thrombosis and haemostasis, vol. 12, no. 11, p. 1874-1879
Publication date2014
Abstract

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 or γ results in the absence of a functional coagulation protein. A patient with congenital afibrinogenemia, due to a FGA homozygous gene deletion, underwent an orthotopic liver transplantation that resulted in complete restoration of normal hemostasis. The patient's explanted liver provided a unique opportunity to further investigate a potential novel treatment modality.

Citation (ISO format)
STROKA, Deborah et al. In vitro rescue of FGA deletion by lentiviral transduction of afibrinogenemic patient’s hepatocytes. In: Journal of thrombosis and haemostasis, 2014, vol. 12, n° 11, p. 1874–1879. doi: 10.1111/jth.12714
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ISSN of the journal1538-7836
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Creation09/09/2014 2:00:00 PM
First validation09/09/2014 2:00:00 PM
Update time03/14/2023 9:47:14 PM
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