Article
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy.
Blood - 15 Apr 2002
Mount Jane D, Herzog Roland W, Tillson D Michael, Goodman Susan A, Robinson Nancy, McCleland Mark L, Bellinger Dwight, Nichols Timothy C, Arruda Valder R, Lothrop Clinton D, High Katherine A
Abstract excerpt
Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation factor IX (FIX). Using adeno-associated virus (AAV)-mediated, liver-directed gene therapy, we achieved long-term (> 17 months) substantial correction of canine hemophilia B in 3 of 4 animals, including 2 dogs wit...
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