Article
Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector.
Human gene therapy - 1 Jul 2005
Brunetti-Pierri Nicola, Nichols Timothy C, McCorquodale Stephanie, Merricks Elizabeth, Palmer Donna J, Beaudet Arthur L, Ng Philip
Abstract excerpt
We have evaluated the potential of liver-directed, helper-dependent adenoviral (HDAd) vector-mediated gene therapy in the hemophilia B dog. Two dogs were injected intravenously with HDAd (3 x 10(12) VP/kg) bearing a liver-restricted canine coagulation factor IX (FIX) expression cassette. After injection, the whole blood clotting time for both dogs declined from >60 min to </=20 min for at least 604 and 446 days,...
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