Article
Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice.
Proceedings of the National Academy of Sciences of the United States of America - 31 Aug 1999
VandenDriessche T, Vanslembrouck V, Goovaerts I, Zwinnen H, Vanderhaeghen M L, Collen D, Chuah M K
Abstract excerpt
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. Improved retroviral vectors expressing high levels of human FVIII were pseudotyped with the vesicular stomatitis virus G...
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