Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

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.

doi:10.1182/blood.v99.8.2670

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

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 …