Defenestrated endothelium delays liver-directed gene transfer in hemophilia A mice.
Sign Up to like & getrecommendations! Published in 2022 at "Blood advances"
DOI: 10.1182/bloodadvances.2021006388
Abstract: Hemophilia A is an inherited bleeding disorder caused by defective or deficient coagulation factor-VIII (FVIII) activity. Until recently, the only treatment for prevention of bleeding involved intravenous administration of FVIII. Gene therapy with adeno associated… read more here.
Keywords: mice; directed gene; gene; liver directed ... See more keywords