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Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

Hemophilia A is caused by a deficiency in coagulation factor VIII (FVIII) and predisposes to spontaneous bleeding that can be life-threatening or lead to chronic disabilities. It is well suited for gene therapy because a moderate increase in plasma FVIII concentration has therapeutic effects. Improv...

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Detalhes bibliográficos
Main Authors: VandenDriessche, Thierry, Vanslembrouck, Veerle, Goovaerts, Inge, Zwinnen, Hans, Vanderhaeghen, Marie-Line, Collen, Desire, Chuah, Marinee K. L.
Formato: Artigo
Idioma:Inglês
Publicado em: The National Academy of Sciences 1999
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC17896/
https://ncbi.nlm.nih.gov/pubmed/10468616
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